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ORPH

ORPH US Stock
$8.75
Open: $8.91 High: $9.105 Low: $8.348 Close: $8.56
Range: 2021-05-06 - 2021-05-07
Volume: 5,364
Market: Closed
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ORPH
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ORPH News
Latest news about the ORPH
  • Orphazyme to present at upcoming investor conferences

    Orphazyme A/SInvestor news No. 05/2021Company Registration No. 32266355 Copenhagen, Denmark and Chicago, IL, USA, April 27, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of neurodegenerative rare diseases, today announced that company management will be participating at the following virtual investor conferences: Redeye Orphan Drugs Seminar Wednesday, April 28, 2021 at 4:15 am EDT/10:15 am CETCorporate presentation by Christophe Bourdon, CEO and Anders Vadsholt, CFOWebcast link: https://www.redeye.se/events/802433/theme-orphan-apr-28th B. Riley Neurosciences ConferenceThursday, April 29, 2021 at 11:00 am EDT/5:00 pm CETCorporate presentation by Christophe Bourdon, CEO and Anders Vadsholt, CFOWebcast link: https://b-riley-neuroscience-conference.events.issuerdirect.com/ For additional information, please contactOrphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 05-2021 Orphazyme to present at upcoming investor conferences

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  • New long-term share-based incentive program

    Orphazyme A/SCompany announcement No. 13/2021Company Registration No. 32266355 Copenhagen, Denmark, April 22, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of neurodegenerative rare diseases, has today introduced a new long-term incentive program (the “LTIP”). The LTIP is designed and structured around the concept of retaining members of the Executive Management and other employees of the Group, while also creating an incentive for a positive share price development and corporate performance for the benefit of the Company’s shareholders. The LTIP grants comprise Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) which entitle the participants, subject to vesting occurring, to be allocated a number of shares in the Company, equivalent to the number of vested RSUs and/or PSUs, against payment of the par value of each share. The RSUs will have a total vesting period of three years calculated from January 1 or July 1 in the grant year and with one third of the granted RSUs vesting on each January 1 or July 1 in the following three financial years. Vesting of RSUs is not conditional upon achieving any financial or non-financial targets. However, vesting is conditional upon (i) the participant remaining employed with a group member throughout the total vesting period for RSUs or the participant becoming a good leaver during the total vesting period for RSUs, in which case the participant will be entitled to keep any vested RSUs and receive a pro rata allocation, and (ii) the participant having complied in all respects with the general terms and conditions as determined by the Board of Directors. The vested RSUs can only be exercised within four months after the expiration of the total vesting period for RSUs. However, the RSU delivery period may be extended to the next open trading window in certain circumstances. The PSUs will have a total vesting period of three years calculated from January 1 or July 1 in the grant year and with the granted PSUs vesting, in whole or in part, on January 1 or July 1 in the third year following the date of the grant. Vesting of PSUs is conditional upon (i) an increase in the quoted share price of the Company’s shares, (ii) the participant remaining employed with a group member throughout the vesting period for PSUs or the participant becoming a good leaver during the vesting period for PSUs, in which case the participant may be entitled to keep a proportion of the PSUs, and (iii) the participant having complied in all respects with the general terms and conditions as determined by the Board of Directors. Any vested PSUs can only be exercised within four months after the expiration of the vesting period for PSUs. However, the PSU delivery period may be extended to the next open trading window in certain circumstances. Based on the current number of participants in the LTIP, the program and other share-based retention grants are expected to comprise up to 950,000 shares in total. The theoretical fair value of each RSU has been estimated at DKK 58.04 and the theoretical fair value of each PSU under the LTIP has been estimated at DKK 20.02. For additional information, please contact Orphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 13-2021 New long-term share-based incentive program

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  • CytRx Notes Orphazyme’s Announcement of Topline Results from Trial for Arimoclomol in the Treatment of Inclusion Body Myositis

    CytRx Corporation (OTCQB: CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ: ORPH) ("Orphazyme") announced that its phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis ("IBM") did not meet its primary and secondary endpoints. According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.

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  • Why Is Orphazyme (ORPH) Stock Trading Lower On Monday?

    Orphazyme A S (NASDAQ: ORPH) saw its stock plunge on Monday after failing to meet primary and secondary endpoints in a Phase 2/3 trial. The experimental drug, arimoclomol, was being tested in 150 inclusion body myositis patients against a placebo, a muscle-wasting disease. The primary objective was to see how well the treatment could prevent disease progression measured by the inclusion body myositis functional rating scale. No significant safety concerns were detected but gave no more data. Full findings from the study will be “shared in a future scientific forum,” the company added in a statement. The drug is designed to boost the production of heat shock proteins, which can “rescue” defective misfolded proteins, clear protein aggregates, and improve lysosomes’ function. This is certainly not the end of the road for arimoclomol. A Phase 3 data readout of the drug in amyotrophic lateral sclerosis, a neurodegenerative disease, is expected in the coming weeks. Arimoclomol is also under a review at the FDA for Niemann-Pick disease type C, a rare disorder in which cholesterol builds up in patients’ cells and leads to neurological problems, with a decision expected by late June. Price Action: ORPH shares are down 29.5% at $8.74 in market trading hours on the last check Monday. See more from BenzingaClick here for options trades from BenzingaGlaxo To Fill About 60M Novavax COVID-19 Shots For BritainIncyte's Pemazyre Wins European Approval For Bile Duct Cancer© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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  • Why Orphazyme's Stock is Down During Today's Session

    Why Orphazyme's Stock is Down During Today's Session The Price And Volume Action In Orphazyme's Stock Today Orphazyme (NASDAQ:ORPH) is currently down 30.59% to a price of $8.75. The stock's current volume for the day is 327.17 thousand, which is approximately 5917.33% of its previous 30-day average volume of 5.53 thousand. Why It's Moving Benzinga Pro's real-time breaking news service shows some insight into what is affecting shares: Orphazyme shares are trading lower after the company's phase 2/3 trial evaluating armioclomol for treatment of inclusion body myositis did not meet its primary and secondary endpoints. Further Analysis of Recent Price Action The stock's 50-day moving average was $12.72 at the time this article was published. Over the past fifty-two weeks, the price has risen as high as $14.78 and fallen to a low of $8.89. Benzinga Pro provides investors with timely and accurate explanations of why a stock is moving. Subscribe to receive real-time alerts explaining stock price movements. Click here to learn more. See more from BenzingaClick here for options trades from BenzingaStocks That Hit 52-Week Lows On Monday© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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  • Orphazyme announces topline results from pivotal trial of arimoclomol for Inclusion Body Myositis (IBM)

    Orphazyme A/SCompany announcement No. 12/2021Inside informationCompany Registration No. 32266355 Copenhagen – March 29, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of neurodegenerative rare diseases, today announced its phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM), a progressively debilitating muscle-wasting disease, did not meet its primary and secondary endpoints. The primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale (IBMFRS). The randomized, placebo-controlled trial was conducted among 150 IBM patients at 12 sites in North America and Europe, in partnership with University College of London and the University of Kansas. Participants were randomized (1:1 ratio) to receive either arimoclomol citrate (400 mg three times daily) or placebo for up to 20 months. No important safety concerns were detected in the trial. The analysis of the data is continuing and complete findings from the study will be shared in a future scientific forum. “We recognize these data are disappointing for patients and families who continue to eagerly await a promising option for IBM. We believe the data collected will be useful to the community, since this trial represents one of the largest, long-term studies ever conducted in this disease and will help inform future research in the category,” said Thomas Blaettler, MD, Chief Medical Officer, Orphazyme. “We are grateful to the investigators and their sites, and the many patients and families who graciously participated in the trial.” “We continue to believe in the promise of arimoclomol and heat shock protein science and are fully committed to our mission to deliver new therapies to patients,” said Anders Vadsholt, interim Chief Executive Officer and Chief Financial Officer, Orphazyme. Orphazyme expects data from a pivotal Phase 3 trial of arimoclomol in Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease, this spring. The company’s applications for arimoclomol (to be branded MIPLYFFATM1) for Niemann-Pick disease type C (NPC) are under priority review with the U.S. Food and Drug Administration, with an expected action date in June 2021, as well as with the European Medicines Agency, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected later this year. For additional information, please contact: Orphazyme A/S Copenhagen: Anders Vadsholt, Interim CEO and CFO +45 28989055 Chicago: Molly Carey Poarch, Global Media +1-773-770-6888 About Orphazyme A/SOrphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for three orphan diseases: Niemann-Pick disease type C (NPC), for which we have a PDUFA date of June 17, 2021, Amyotrophic Lateral Sclerosis (ALS), and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA.CO). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in ten phase 1, four phase 2 and two pivotal phase 2/3 trials. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. About IBMInclusion Body Myositis (IBM) is a progressively debilitating muscle-wasting disease. IBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability. There are no approved treatments for IBM. Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of IBM. Forward-looking statementThis company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. 1 MIPLYFFA is a trademark or registered trademark of Orphazyme A/S. The global brand “MIPLYFFA” has received conditional approval from the U.S. Food and Drug Administration; the brand name will be used commercially upon approval for NPC. Attachment 12-2021 Orphazyme announces topline results from pivotal trial of arimoclomol for Inclusion Body Myositis (IBM)

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  • Reporting of transactions in Orphazyme’s shares made by persons discharging managerial responsibilities

    Orphazyme A/SCompany announcement No. 11/2021 Company Registration No. 32266355 Copenhagen, Denmark, March 25, 2021 – Pursuant to the Market Abuse Regulation article 19, Orphazyme A/S, CVR no. 32266355 (“Orphazyme”), hereby notifies receipt of information of the following transactions in Orphazyme’s shares by persons discharging managerial responsibilities in Orphazyme: 1 Details of the Reporting Person / Closely Associated Persona) NameCatherine Moukheibir2 Reason for the notification a) Position/statusMember of the Board of Directorsb) Initial notification/AmendmentInitial notification3 Details of the Company, emission allowance market participant, auction platform, auctioneer or auction monitor a) NameOrphazyme A/Sb) LEI code54930025OZD2GGSQ7L424 Details of the transaction(s): section to be repeated for (i) each type of instrument; (ii) each type of transaction; (iii) each date; and (iv) each place where transactions have been conducted a) Description of the financial instrument, type of instrument and identification codeSharesISIN DK0060910917b) Nature of the transactionExercise of Restricted Share Unitsc) Price(s) and volume(s) Price(s) Volume(s) DKK61.18951,927 d) Aggregated informationAggregated volume: 1,927 sharesAggregated price: DKK 117,912.17Price per share (volume weighted average): DKK 61.1895e) Date of the transactionMarch 24, 2021f) Place of the transactionOutside a trading venue 1 Details of the Reporting Person / Closely Associated Persona) NameMartijn Kleijwegt2 Reason for the notification a) Position/statusMember of the Board of Directorsb) Initial notification/AmendmentInitial notification3 Details of the Company, emission allowance market participant, auction platform, auctioneer or auction monitor a) NameOrphazyme A/Sb) LEI code54930025OZD2GGSQ7L424 Details of the transaction(s): section to be repeated for (i) each type of instrument; (ii) each type of transaction; (iii) each date; and (iv) each place where transactions have been conducted a) Description of the financial instrument, type of instrument and identification codeSharesISIN DK0060910917b) Nature of the transactionExercise of Restricted Share Unitsc) Price(s) and volume(s) Price(s) Volume(s) DKK61.18951,927 d) Aggregated informationAggregated volume: 1,927 sharesAggregated price: DKK 117,912.17Price per share (volume weighted average): DKK 61.1895e) Date of the transactionMarch 24, 2021f) Place of the transactionOutside a trading venue 4 Details of the transaction(s): section to be repeated for (i) each type of instrument; (ii) each type of transaction; (iii) each date; and (iv) each place where transactions have been conducted a) Description of the financial instrument, type of instrument and identification codeSharesISIN DK0060910917b) Nature of the transactionSale of shares to LSP V Cöoperatieve U.A.c) Price(s) and volume(s) Price(s) Volume(s) EUR-1,927 d) Aggregated informationAggregated volume: 1,927 sharesAggregated price: EUR 1Price per share (volume weighted average): EUR - e) Date of the transactionMarch 24, 2021f) Place of the transactionOutside a trading venue 1 Details of the Reporting Person / Closely Associated Persona) NameLSP V Cöoperatieve U.A.2 Reason for the notification a) Position/statusClosely Associated Person to Martijn Kleijwegt, member of the Board of Directorsb) Initial notification/AmendmentInitial notification3 Details of the Company, emission allowance market participant, auction platform, auctioneer or auction monitor a) NameOrphazyme A/Sb) LEI code54930025OZD2GGSQ7L424 Details of the transaction(s): section to be repeated for (i) each type of instrument; (ii) each type of transaction; (iii) each date; and (iv) each place where transactions have been conducted a) Description of the financial instrument, type of instrument and identification codeSharesISIN DK0060910917b) Nature of the transactionPurchase of shares from Martijn Kleijwegtc) Price(s) and volume(s) Price(s) Volume(s)EUR-1,927 d) Aggregated informationAggregated volume: 1,927 sharesAggregated price: EUR 1Price per share (volume weighted average): EUR - e) Date of the transactionMarch 24, 2021f) Place of the transactionOutside a trading venue For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO & CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 11-2021 Reporting of transactions in Orphazyme’s shares made by persons discharging managerial responsibilities

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  • Articles of Association for Orphazyme A/S

    Orphazyme A/SCompany announcement No. 10/2021 Company Registration No. 32266355 Copenhagen, Denmark, March 25, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“the Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today held its Annual General Meeting, at which amendments to the Articles of Association of the Company were adopted. The new Articles of Association dated 25 March 2021 are attached and are available on the Company’s website www.orphazyme.com. For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO & CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachments 10-2021 Articles of Association for Orphazyme Orphazyme - Articles of Assocation - 25 March 2021 Orphazyme - Vedtægter - 25. marts 2021

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  • Capital increase of 3,854 shares in Orphazyme A/S as a result of the exercise of Restricted Share Units

    Orphazyme A/SCompany announcement No. 09/2021 Company Registration No. 32266355 Copenhagen, Denmark, March 25, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“the Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, announces that a share capital increase has been registered with the Danish Business Authority as a result of the exercise of Restricted Share Units by board members. A total of 3,854 shares of nominally DKK 1 each have been issued by the Company at a subscription price per Restricted Share Unit of DKK 61.1895. Following the share capital increase, the total nominal share capital will be DKK 34,952,241, divided into 34,952,241 shares each with a nominal value of DKK 1. Each share carries one vote at Orphazyme's general meetings and accordingly the total number of voting rights of the company are 34,952,241. Updated Articles of Association can be found at www.orphazyme.com. For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO & CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 09-2021 Capital increase of 3,854 shares in Orphazyme as a result of the exercise of Restricted Share Units

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  • Resolutions passed at the Annual General Meeting

    Orphazyme A/SCompany announcement No. 08/2021 Company Registration No. 32266355 Copenhagen, Denmark, March 25, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (the “Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today held its Annual General Meeting, at which the Annual General Meeting: Took note of the Board of Directors’ report on the Company’s activities in the past financial year;Adopted the Company’s Annual Report 2020;Adopted that the loss related to the financial year 2020 is carried forward;Granted discharge of liability to the Board of Directors and the Executive Management in relation to the Annual Report 2020;Approved the remuneration report for 2020 in the advisory vote;Approved the remuneration of the Board of Directors for the current financial year;Re-elected Georges Gemayel, Bo Jesper Hansen, Anders Hedegaard, Carrolee Barlow, Catherine Moukheibir, Martijn Kleijwegt, Martin Bonde, and Rémi Droller as members of the Board of Directors;Elected Stephanie Okey as new member of the Board of Directors;Re-elected EY Godkendt Revisionspartnerselskab as the Company’s auditor in accordance with the recommendation from the Audit Committee;Adopted an authorization to the Board of Directors to approve the acquisition of treasury shares in the period until March 25, 2026 with a total nominal value of up to 10% of the share capital of the Company subject to the Company’s holding of treasury shares after such acquisition does not exceed 20% of the Company’s share capital;Approved certain adjustments to the Company’s Remuneration Policy, including (i) adjustments to allow for a share-based incentive program to the Executive Management and certain employees comprising restricted share units and performance share units, (ii) adjustment of the shareholding requirement for the Executive Management, (iii) adjustment of the claw back clause with the purpose of aligning the clause with the revised Danish Recommendations on Corporate Governance and (iv) adjustment of the situations under which accelerated vesting may occur;Adopted an amendment to Article 3.1 of the Articles of Association regarding renewal and extension of the existing authorization to the Board of Directors to increase the Company’s share capital without pre-emption rights for existing shareholders by up to a nominal amount of DKK 6,989,767 and the deletion of Article 3.1.1; andAdopted an authorization to the Board of Directors included in a new Article 3.5 of the Articles of Association to increase the share capital in the period until March 25, 2026 without pre-emption rights for existing shareholders by up to a nominal amount of DKK 1,300,000 in connection with the issue of new shares to members of the Board of Directors, executives and/or employees of the Company subject to a total cap of nominally DKK 2,000,000 for both authorizations under the current Article 3.2 and the new Article 3.5 of the Articles of Association. After the Company’s Annual General Meeting was held, the Board of Directors constituted itself by appointing Georges Gemayel as Chairman and Bo Jesper Hansen as Deputy Chairman of the Board of Directors. For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO and CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM, and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 08-2021 Resolutions passed at the Annual General Meeting

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  • Correction: Notice to convene Annual General Meeting

    Orphazyme A/SCompany announcement No. 07/2021 Company Registration No. 32266355 [Correction: Hereby including all relevant attachments] Copenhagen, Denmark, March 3, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (the “Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced that the Company’s Annual General Meeting will be held on: Thursday, March 25, 2021 at 5:00 PM (CET) at the Company’s address Ole Maaløes Vej 3, DK-2200 Copenhagen N, Denmark. In order to protect the health and safety of all, reduce the risk of COVID-19 spreading, and given the Danish government’s restrictions on assembly, we strongly recommend that shareholders refrain from attending the Annual General Meeting in person and instead exercise their shareholder rights by giving proxy to the Board of Directors or by voting by correspondence prior to the Annual General Meeting. The notice to convene the Annual General Meeting, including Appendix 1: Candidates for the Board of Directors and Appendix 2: Revised Remuneration Policy, is attached. Additional information about the Annual General Meeting is available on the Company’s website:https://orphazyme.gcs-web.com/annual-general-meeting-0 For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO and CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachments 07-2021 Notice to convene Annual General Meeting Notice to convene Annual General Meeting Appendix 1 - Candidates for the Board of Directors Appendix 2 - Revised Remuneration Policy

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  • Notice to convene Annual General Meeting

    Orphazyme A/SCompany announcement No. 07/2021 Company Registration No. 32266355 Copenhagen, Denmark, March 3, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (the “Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced that the Company’s Annual General Meeting will be held on: Thursday, March 25, 2021 at 5:00 PM (CET) at the Company’s address Ole Maaløes Vej 3, DK-2200 Copenhagen N, Denmark. In order to protect the health and safety of all, reduce the risk of COVID-19 spreading, and given the Danish government’s restrictions on assembly, we strongly recommend that shareholders refrain from attending the Annual General Meeting in person and instead exercise their shareholder rights by giving proxy to the Board of Directors or by voting by correspondence prior to the Annual General Meeting. The notice to convene the Annual General Meeting, including Appendix 1: Candidates for the Board of Directors and Appendix 2: Revised Remuneration Policy, is attached. Additional information about the Annual General Meeting is available on the Company’s website:https://orphazyme.gcs-web.com/annual-general-meeting-0 For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO and CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachments 07-2021 Notice to convene Annual General Meeting Appendix 1 - Candidates for the Board of Directors Appendix 2 - Revised Remuneration Policy

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  • Orphazyme reports business highlights and financial results in Annual Report 2020

    Orphazyme A/SCompany announcementNo. 06/2021Company Registration No. 32266355 -Advanced arimoclomol in 4 rare disease indications; announced global brand name of arimoclomol (MIPLYFFATM1); PDUFA date of June 17, 2021 for NPC; MAA under review with EMA- -Completed successful $88M IPO in US on Nasdaq; DKK 1.3 billion (USD ~200 million) raised in 2020 will support potential commercialization- -Expanded U.S. and European footprint to support commercial launch readiness- Copenhagen, Denmark, March 2, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“the Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative rare diseases, today reports its business highlights and financial results included in its Annual Report for the period from January 1, 2020 to December 31, 2020. “2020 was an important year for Orphazyme. We invested in all aspects of our business to prepare to bring our investigational Heat-Shock Protein response therapy, arimoclomol, to people living with neurodegenerative, rare diseases,” said Anders Vadsholt, Interim Chief Executive Officer and Chief Financial Officer of Orphazyme. “2021 is shaping up to be transformative for Orphazyme. We expect to execute on several important goals, including the U.S. Food and Drug Administration’s (FDA) potential approval of arimoclomol in our first indication, Niemann-Pick disease Type C (NPC), and reporting results from late-stage, pivotal, clinical trials of arimoclomol in Amyotrophic Lateral Sclerosis (ALS) and Inclusion Body Myositis (IBM). With the completion of our global offering, including our U.S. initial public offering (IPO) of American Depositary Shares in the U.S., in 2020, and the appointment of Christophe Bourdon as Chief Executive Officer as of April 1, 2021, we are operating with a strong financial and commercial position and believe we are well positioned to advance Orphazyme into a global, commercial-stage company and create value for our shareholders.” 2020 arimoclomol highlights Accelerated commercial and other pre-launch activities in preparation for potential approval of arimoclomol in NPCReceived FDA acceptance, with Priority Review, of our New Drug Application (NDA) for arimoclomol for the treatment of NPCSubmitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for arimoclomol for the treatment of NPCAnnounced a U.S. Early Access Program for arimoclomol for treatment of NPC, with 10 sites across the U.S. at year-end 2020 ·Received U.S. Fast-Track Designation for arimoclomol in ALS ·Reported top-line data from a Phase 2 clinical trial in Gaucher disease Type 1 and Type 3 demonstrating marked improvements in key clinical markers following six months of treatment with arimoclomol 2020 business highlights Completed a global offering, including our IPO of American Depositary Shares on The Nasdaq Global Select Market in the U.S., raising gross proceeds from the issuance of new ordinary shares (including ordinary shares represented by American Depositary Shares) of DKK 534 million (USD 88 million)Completed a directed issue and private placement of DKK 745 million (USD 112 million), bringing total gross capital raised in 2020 to DKK 1.3 billionEstablished U.S. main office in Chicago in preparation for commercialization of arimoclomolInitiated a search for the next Chief Executive Officer following the resignation of Kim Stratton in December 2020 Subsequent 2021 events Presented data supporting the profile of arimoclomol at the virtual WORLDSymposium in February 2021Announced global brand name for arimoclomol, MIPLYFFATM1Appointed Christophe Bourdon as new Chief Executive Officer as of April 1, 2021 Upcoming events 2021 Potential approval of arimoclomol in the U.S. and EU for the treatment of NPC; PDUFA target date in the U.S. of June 17, 2021; expected decisions from the European regulators in Q4 2021Topline data read-outs from pivotal studies of arimoclomol in ALS and IBM in H1 2021 2020 financial results For the period January 1, 2020 to December 31, 2020: Net loss amounted to DKK 633.2 million compared to a net loss of DKK 337.5 million for the same period in 2019. The increased net loss was primarily driven by a continued investment in research and development activities, escalation of our commercial launch preparations and strengthening of our global teamResearch and development expenses for the period totaled DKK 361.3 million compared to DKK 285.4 million for the same period in 2019. The increase of DKK 75.9 million was mainly attributable to costs related to three clinical pharmacology registrational trials that ramped up and took place mainly during 2020 in addition to increased clinical safety reporting activity in the ongoing clinical trials. In addition, our employee costs increased as a result of 12 more research and development employees hired during 2020General and administrative expenses for the period totaled DKK 247.3 million compared to DKK 50.5 million for the same period in 2019. The increase of DKK 196.7 million was primarily due to the build-up of our commercial organization as well as expenses related to increased expenses for being a listed company in the U.S.As of December 31, 2020, Orphazyme held cash totaling DKK 726.9 million compared to DKK 123.6 million as of December 31, 2019 2021 outlook For 2021, Orphazyme anticipates an operating loss in the range of DKK 100 - 150 million, operating expenses in the range DKK 800 – 850 million and a cash position at year-end 2020 of more than DKK 350 million. Our 2021 outlook for operating loss, operating expenses, and cash position includes the following key assumptions: The approval of arimoclomol for NPC in the U.S. by the PDUFA action date of June 17, 2021;The grant of a Priority Review Voucher (PRV) upon such approval and our ability to sell the PRV voucher at generally-accepted market rates;Initial revenues from arimoclomol in the U.S. and named patient sales in certain countries;Approval of arimoclomol for NPC in Europe in Q4 2021;A gradual increase in arimoclomol sales through the second half of 2021, reaching DKK 60 - 120 million (~ USD 10 - 20 million) in revenues by year-end;Continued investments in our commercial infrastructure in the U.S. and Europe to support product launches;R&D expenses to support the advancement and completion of arimoclomol clinical trials in IBM and ALS, including data readout and preparations for filing, if successful Further details on our 2021 outlook are outlined in the Annual Report 2020, published March 2, 2021. The full report is attached as a PDF file and can be found on the Company's global website at www.orphazyme.com. Webcast and conference call Orphazyme will be hosting an investor call during which the Orphazyme management team will be presenting the Company’s 2020 Annual Report. The presentation will be followed by a Q&A session. The call will be held on Tuesday, March 2, 2021 at 2.00 PM CET / 8.00 AM EST. Dial-in details: ·Denmark: +45 32720417 ·France: +33 (0) 170700781 ·Netherlands: +31 (0) 207956614 ·Standard International: +44 (0) 2071 928338 ·Sweden: +46 (0) 856618467 ·United Kingdom: +44 (0) 8444819752 ·United States: +1 6467413167 Event Title: Orphazyme Annual Report 2020 Confirmation code: 8239516 The presentation will also be available via webcast: https://edge.media-server.com/mmc/p/kmp575pf After the call, the presentation will be available via the above link. For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO and CFO +45 28 98 90 55Molly Carey Poarch, Global Communications +1 773-770-6888 1. MIPLYFFA is a trademark or registered trademark of Orphazyme A/S. The global brand “MIPLYFFA” has received conditional approval from the U.S. Food and Drug Administration; the brand name will be considered approved for commercial use with the approval for NPC. About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein (HSP) response for the treatment of neurodegenerative rare diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of HSPs. HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the U.S. and EU. Arimoclomol has received fast-track designation (FTD) from the FDA for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. About NPCNiemann-Pick disease type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a family known as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids that are normally cleared by the lysosome accumulate in tissues and organs, including the brain, and drive the disease pathology. We estimate the incidence of NPC to be one in 100,000 live births and the number of NPC patients in the United States and in Europe to be approximately 1,800 individuals. There are no approved treatments for NPC in the U.S. About IBMInclusion Body Myositis (IBM) is a progressively debilitating muscle-wasting disease. IBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability. The estimated prevalence of IBM is 24.8-45.6 per million or 17,000-31,000 patients in the U.S. and Europe. There are no approved treatments for IBM. Arimoclomol has been granted Orphan Drug Designation (EU and U.S.) for the treatment of IBM. About ALS Amyotrophic Lateral Sclerosis (ALS) is a rare, rapidly progressive, and always fatal neurodegenerative disease. Protein misfolding and aggregation in motor neurons are important contributors to the disease process, which ultimately leads to paralysis of skeletal muscles as well as the muscles that enable breathing. The patient population in Europe and the United States is estimated to be approximately 50,000 patients. Currently, there are only limited treatment options available. Arimoclomol has been granted Orphan Drug Designation (EU and U.S.) for the treatment of ALS. About GaucherGaucher disease is a rare, inherited metabolic disorder causing certain sugar containing fats to abnormally accumulate in the lysosomes of cells, especially within cells of the blood system and nerve cells, thereby affecting organs such as the brain, bone marrow, spleen and liver. The typical systemic symptoms of Gaucher disease, which can appear at any age, include an abnormally enlarged liver and/or spleen and low levels of circulating red blood cells and platelets. These systemic symptoms can be treated by existing enzyme replacement therapy (ERT), and substrate reduction therapy (SRT). The neurological symptoms, although heterogeneous, may include muscle rigidity, loss of movement, seizures, cognitive impairment and vision problems and are unable to be treated by these therapies, given their inability to cross the blood brain barrier (BBB). Gaucher disease is the most common lysosomal storage disorder (LSD) with an estimated incidence of 1:40,000 to 1:60,000, and affecting approximately 15,000 individuals in the United States and Europe combined. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachments 06-2021 Orphazyme reports business highlights and financial results in Annual Report 2020 Annual Report 2020 Remuneration Report 2020

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  • Orphazyme appoints Christophe Bourdon as Chief Executive Officer

    Orphazyme A/SCompany announcement No. 05/2021Inside informationCompany Registration No. 32266355 Copenhagen, Denmark, March 1, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced the appointment of Christophe Bourdon as the company’s Chief Executive Officer, effective as of April 1, 2021, following approval from the Board of Directors. Christophe Bourdon has a strong track record in launching and commercializing rare and non-rare disease products in Europe and the United States. He has a deep knowledge of patient journey, market access, and payer dynamics on both sides of the Atlantic. During his 25 years in the biotech/pharmaceutical industry working in three continents and holding several leadership positions, Christophe initiated and accelerated significant strategic transformations and has proven experience building successful multi-cultural and cross-functional teams. He is adept at fostering an environment focused on innovation with one constant driver in mind: Making a meaningful difference for patients. He successfully launched a variety of products in demanding environments, making him an ideal candidate to lead Orphazyme as it prepares for a potential commercial launch of its investigational product candidate, arimoclomol, in the United States and Europe. Christophe Bourdon comes to Orphazyme from his position with Amgen, Inc. as Senior Vice President, General Manager, U.S. Oncology Business, where he led commercialization planning and execution for several products. Prior to Amgen, Christophe was Senior Vice President of Europe, Middle East, Africa, and Canada at Alexion as the company launched two breakthrough ultra-orphan drugs and negotiated payor access across UK, Germany, France, Italy, and Canada. Christophe Bourdon holds an MBA from IMD business school (Switzerland) and a BA from ISG (France). Chairman of the Board of Directors, Georges Gemayel, states “I am delighted to announce Christophe Bourdon will join our leadership team as Chief Executive Officer starting April 1, 2021. This is an important time for Orphazyme, with numerous near-term milestones that will shape the company’s future direction. Our Board conducted an extensive search for a leader who can guide not only our near-term execution, but also align the company around a vision for impact and scale for our long-term growth ambitions. With his strong track record on growth over two decades in relevant roles, Mr. Bourdon will bring his dynamic leadership and skillset to successfully build out and integrate our organization as we prepare for the anticipated commercial launch of our investigational product candidate, arimoclomol, in the United States and Europe. As an authentic and result-oriented leader, he believes in building a transparent and inclusive corporate culture and is the right choice to lead Orphazyme into its next growth phase.” Christophe Bourdon states “The opportunity to join Orphazyme was compelling for me, not only based on its purposeful mission, but also the incredible near-term opportunities to create impact for patients. It is both exciting and humbling to assume this role at such a pivotal time,” said Mr. Bourdon. “Building on the learnings I have gained from my invaluable experience at Amgen and the rare disease experience garnered at Alexion, I look forward to championing this talented team to advance the mission on behalf of our patient communities and deliver value for our shareholders. I am also very much looking forward to relocating to Copenhagen.” For additional information, please contact Orphazyme A/S Anders Vadsholt, Interim CEO and CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. About NPCNiemann-Pick disease Type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a family known as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids that are normally cleared by the lysosome accumulate in tissues and organs, including the brain, and drive the disease pathology. We estimate the incidence of NPC to be one in 100,000 live births and the number of NPC patients in the United States and in Europe to be approximately 1,800 individuals. There are no approved treatments for NPC in the U.S. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 05-2021 Orphazyme appoints Christophe Bourdon as Chief Executive Officer

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  • Orphazyme announces participation in upcoming virtual investor conferences

    Orphazyme A/SInvestor news No. 04/2021 Company Registration No. 32266355 Copenhagen, Denmark and Chicago, IL, February 26, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“the Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative rare diseases, today announced that Anders Vadsholt, interim CEO and CFO of Orphazyme, will be participating in the following upcoming investor conferences: Cowen Healthcare Conference, March 1-3, 2021: Panel discussion moderated by Cowen analysts Phil Nadeau, Ritu Baral and Boris Peaker entitled “New Drug Launches Panel” on Wednesday, March 3, 2021 from 10:20-11:20 AM EST Guggenheim Healthcare Talks, 2021 Genomic Medicines & Rare Diseases Conference, April 1, 2021: Orphazyme will participate in 1x1s and small group meetings with investors A live webcast of the panel will be available on the Orphazyme global website under the Investor Relations section. For additional information, please contactOrphazyme A/S Anders Vadsholt, Interim CEO and CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative rare diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), sporadic Inclusion Body Myositis (sIBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, sIBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, sIBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 04-2021 Orphazyme announces participation in upcoming virtual investor conferences

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  • Capital increase of 80,553 shares (equivalent to approximately 0.23% of the existing shares) in Orphazyme A/S as a result of an issue of bonus shares to KUMCRI and directed issuance of new shares

    Orphazyme A/SCompany announcement No. 04/2021 Company Registration No. 32266355 Copenhagen, Denmark, February 25, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“the Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today decided on a capital increase as a result of a directed issue of bonus shares to the University of Kansas Medical Center Research Institute, Inc. (“KUMCRI”) and a directed issue of new shares to Kim Stratton, former CEO of the Company. The capital increase was decided pursuant to the Board of Directors’ authorization laid down in Article 3.2 and 3.3 of the Articles of Association. According to the license agreement entered into on October 31, 2017 between among others the Company and KUMCRI, the Company shall, following the end of each calendar year, issue or otherwise deliver to KUMCRI a total number of shares equal to half of the aggregate amount of cash funding that KUMCRI has reported to have spent under the grant from the US Food and Drug Administration received with respect to the Company’s on-going Phase 2/3 clinical trial on arimoclomol for the treatment of sIBM. On the basis of a financial report of expenditures provided by KUMCRI, the Company will issue 22,553 new shares each with a nominal value of DKK 1, equal to approximately 0.065% of its total share capital, in favor of KUMCRI. Accordingly, the Company’s share capital will be increased by nominally DKK 22,553 by way of an issue of bonus shares at par value by transfer of DKK 22,553 from the Company’s distributable reserves. The number of shares to be delivered to KUMCRI has been calculated based on the average closing price of the Company’s shares on Nasdaq Copenhagen during a 30-day period preceding January 29, 2021. According to agreements entered into between Kim Stratton and the Company, Kim Stratton is entitled to receive a grant of 58,000 shares at a subscription price of DKK 1 per share as the conditions related to the grant have been met. Thus, the Company will issue 58,000 new shares each with a nominal value of DKK 1, and the share capital will be increased by nominally DKK 58,000. Accordingly, the Company will issue a total of 80,553 shares, and the Company’s share capital will in total be increased by nominally DKK 80,553, equal to approximately 0,23% of the Company’s existing share capital. The new shares are negotiable instruments and will in every respect carry the same rights as the existing shares from the time of registration of the capital increase with the Danish Business Authority (which is expected to occur today). The new shares are expected to be admitted to trading and official listing on Nasdaq Copenhagen in the week commencing March 1, 2021. Following the share capital increase, the total nominal share capital will be DKK 34,948,387, divided into 34,948,387 shares each with a nominal value of DKK 1. Each share carries one vote at the Company's general meetings and accordingly the total number of voting rights in the Company will be 34,948,387. Updated Articles of Association can be found at www.orphazyme.com. For additional information, please contact Orphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 04-2021 Capital increase of 80,553 shares as a result of an issue of bonus shares to KUMCRI and directed issuance of new shares

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  • Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

    Orphazyme USInvestor news No. 02/2021 Chicago, USA, February 4, 2021 – Orphazyme US (ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced plans to present a series of data on its investigational drug arimoclomol during the 17th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021. “The WORLDSymposium, while virtual this year, offers an important opportunity to present the mechanisms of the Heat-Shock Protein response and how that translates into potential therapeutic utility for lysosomal diseases such as Niemann-Pick disease Type C (NPC),” said Daniel Gallo, Head, U.S. Medical, Orphazyme. “We look forward to convening with the research community during the conference and to showcasing these data in support of arimoclomol.” Arimoclomol data being presented at the virtual WORLDSymposium are: Persistent effect of arimoclomol in patients with Niemann-Pick disease Type C: 12-month results from an open-label extension of a pivotal phase 2/3 study Presented by Marc Patterson, M.D.Wednesday, February 10, Poster 191 Pharmacokinetics properties of arimoclomol in Niemann-Pick disease Type C: Modest and not clinically relevant effect of bodyweight or age Presented by Thomas AndersonThursday, February 11, Poster 5 Rescue of NPC1 protein by the Heat-Shock response amplifier arimoclomol across multiple genotypes Presented by Nikolaj Petersen, Ph.D.Thursday, February 11, Poster 197 Impacts and burden of NPC: A Patient and Caregiver Perspective Presented by Eugen Mengel, M.D.Thursday, February 11, Poster 158 Validation of a short-form 5-domain Niemann-Pick disease type C clinical severity scale (5-domain NPCCSS) Presented by Marc Patterson, M.D.Thursday, February 11, Poster 190 Arimoclomol is under Priority Review by the U.S. Food and Drug administration (FDA) for the treatment of NPC with a target action date of June 17, 2021. NPC is a rare, progressive, life-threatening, neurodegenerative disease for which there is no cure and no approved treatment in the United States. Arimoclomol is also under review in Europe for the NPC indication. “We continue to engage in productive dialogue with the FDA related to the NDA for arimoclomol,” said Thomas Blaettler, Chief Medical Officer, Orphazyme. “In parallel, we continue to enroll patients in our Early Access Program (EAP) in the US and we are initiating EAPs in European countries, including France and Germany. These activities advance Orphazyme’s mission to bring an innovative treatment to rare disease communities with significant unmet need.” For additional information, please contact Orphazyme US Chicago, USA: Molly Carey Poarch +1 773-770-6888 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. About NPCNiemann-Pick disease Type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a family known as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids that are normally cleared by the lysosome accumulate in tissues and organs, including the brain, and drive the disease pathology. We estimate the incidence of NPC to be one in 100,000 live births and the number of NPC patients in the United States and in Europe to be approximately 1,800 individuals. There are no approved treatments for NPC in the U.S. Forward-looking statement This company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 02-2021 Orphazyme to Showcase Data on Arimoclomol in Niemann-Pick Disease Type C During the 2021 Annual WORLDSymposium™

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  • Capital increase of 170,131 shares in Orphazyme A/S as a result of vesting and exercise of Matching Shares

    Orphazyme A/SCompany announcement No. 02/2021 Company Registration No. 32266355 Copenhagen, Denmark, February 1, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces that a share capital increase has been registered with the Danish Business Authority as a result of the vesting and exercise of Matching Shares. A total of 170,131 shares of nominally DKK 1 each have been issued by the Company at a subscription price per Matching Share of DKK 1. Following the share capital increase, the total nominal share capital will be DKK 34,867,834, divided into 34,867,834 shares each with a nominal value of DKK 1. Each share carries one vote at Orphazyme's general meetings and accordingly the total number of voting rights of the company are 34,867,834. Updated Articles of Association can be found at www.orphazyme.com. For additional information, please contact Orphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. Orphazyme is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the Orphazyme’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), sporadic Inclusion Body Myositis (sIBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, sIBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, sIBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, sIBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statements This company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof . Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 02-2021 Capital increase of 170,131 shares in Orphazyme AS as a result of vesting and exercise of Matching Shares

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  • Major shareholder announcement

    Orphazyme A/SCompany announcement No. 01/2021 Company Registration No. 32266355 Copenhagen, Denmark, January 18, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“the Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, hereby announces the receipt of notification pursuant to Section 38 of the Danish Capital Markets Act from Danske Bank A/S, that as of January 15, 2021, Danske Bank A/S holds a total of 34,697,703 shares in the Company, corresponding to 3.49% of the Company’s share capital and that Danske Bank A/S as of January 15, 2021 controls 4.95% of the voting rights in the Company. Danske Bank A/S’ shareholding consists of a 2.24% indirect and 1.25% direct ownership through Danica Pension Livsforsikringsaktieselskab, Danica Pension Försäkringsaktiebolag, Investeringsforeningen Danske Invest and Danske Invest SICAV. Danske Bank A/S’ control of voting rights in the Company consists of a 3.70% indirect and 1.25% direct control through Danica Pension Livsforsikringsaktieselskab, Danica Pension Försäkringsaktiebolag, Investeringsforeningen Danske Invest and Danske Invest SICAV. For additional information, please contact Orphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/SThe Company is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The Company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the Company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM), and Gaucher disease. The Company is headquartered in Denmark and has operations in the U.S. and Switzerland. The Company’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomolArimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood-brain barrier, and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received Fast Track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM, and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statementsThis company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment 01-2021 Major shareholder announcement

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  • Orphazyme to take part in panel discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event

    Orphazyme A/S Investor news                                                                                                        No. 01/2021                                                                                                           Company Registration No. 32266355 Copenhagen, Denmark, January 4, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces Anders Vadsholt, Chief Financial Officer of Orphazyme, will take part in a panel discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event, which is being held virtually from January 6-8 and 11-14, 2021.The panel is titled “EU Companies IPO-ing in the US: Benefits of EU Companies Listing on NASDAQ” and takes place on Thursday, January 7, 2021 at 10:00 AM Eastern Standard Time. Investors can pre-register for the panel discussion here. For additional information, please contactOrphazyme A/S Anders Vadsholt Chief Financial Officer +45 28 98 90 55 afv@orphazme.comInvestors, EU LifeSci Advisors, LLC Mary-Ann Chang +44 7483 284 853 mchang@lifesciadvisors.comInvestors, US LifeSci Advisors, LLC Ashley R. Robinson +617 775 5956 arr@lifesciadvisors.com About Orphazyme A/S  Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S., Switzerland, France and Germany. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA.CO). About arimoclomol  Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood-brain barrier, and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC.Forward-looking statement  This company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Attachment * 01-2021 Orphazyme to take part in panel discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event

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