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About OrphazymeOrphazyme ApS is a Danish biotech company that develops paradigm-changing medicines for the treatment of protein misfolding diseases with well-characterised mechanisms of disease. The lead programme is in development as a treatment for the lysosomal storage disease Niemann-Pick disease type C. This is one of a family ~50 debilitating genetic disorders that often affect children, most of whom are currently untreatable.
CEO: Anders Hinsby
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27 articles with Orphazyme
Orphazyme A/S, a late-stage biopharmaceutical company, announces its Interim Report First Half 2021 for the period January 1 – June 30, 2021.
Orphazyme said that its OLE trial, which has been running for the past 24 months, showed that arimoclomol could be an effective and safe option in treating Niemann-Pick disease type C.
Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research
Orphazyme A/S, a late-stage biopharmaceutical company, announced 24-month interim results of an open-label extension trial, providing efficacy and safety data for its investigational treatment arimoclomol in Niemann-Pick disease type C for up to 36 months.
Orphazyme announces restructuring to focus resources on supporting a path forward for arimoclomol in NPC
Orphazyme A/S, a late-stage biopharmaceutical company, announced a restructuring intended to enable the company to advance its corporate strategy and the development of arimoclomol for Niemann-Pick disease type C.
Orphazyme A/S hereby announces the receipt of notification pursuant to Section 38 of the Danish Capital Markets Act from Aescap Venture Management B.V, that as of June 16, 2021, Aescap Venture Management B.V indirectly holds shares corresponding to less than 5% of the total share capital and voting rights in the Company.
The FDA said that the drug, which has been studied and submitted for NPC indication, needs further data to support its use in this rare, progressive genetic disorder.
Orphazyme A/S announced it has received a Complete Response Letter from the U.S. Food and Drug Administration following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C.
The month of June continues to be busy for the U.S. Food and Drug Administration. There are three PDUFA dates on the calendar for this week. Here’s a look.
Orphazyme A/S, a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, announced that American Depositary Shares representing its ordinary shares on Nasdaq US have since June 10, 2021 experienced extreme volatility in price and trading volume.
4/5/2021It was a busy week for clinical trial news. Here’s a look.
Orphazyme announced its Phase II/III clinical trial of arimoclomal for inclusion body myositis (IBM) failed to hit both its primary and secondary endpoints.
It will be reviewed under Priority Review with a PDUFA date of March 17, 2021.
CytRx Corporation (OTCQB: CYTR), a biopharmaceutical research and development company specializing principally in oncology and neurodegenerative diseases, today clarified certain prior disclosures regarding its relationship with Orphazyme A/S.
Clinigen and Orphazyme launch an Early Access Program for arimoclomol in patients with Niemann-Pick disease type C
Clinigen Group plc has partnered with Orphazyme A/S, a global biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, to introduce and administer an Early Access Program for arimoclomol in patients with Niemann-Pick disease type C in the US.
Montrium, today announces that Orphazyme, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, has chosen to extend Montrium’s eTMF Connect and RegDocs Connect solutions with Quality Connect to empower end-to-end commercialization processes.
Orphazyme Completes Enrollment In Phase 3 Trial Evaluating Arimoclomol In Amyotrophic Lateral Sclerosis
Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announces that it has completed enrollment in its phase 3 trial evaluating arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS) ahead of schedule.
Orphazyme reports encouraging arimoclomol clinical trial top-line data in Niemann-Pick disease Type C (NPC)
Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced encouraging top-line results for its clinical Phase II/III trial with orally administered arimoclomol for the treatment of patients with Niemann-Pick disease Type C (NPC).
Centogene, the worldwide leader in elucidating rare disease genetics for patients, clinicians and pharmaceutical partners, today announced a clinical development collaboration with Orphazyme A/S.
Capital Increase of 11,380 Shares (Equivalent to Approximately 0.06% of the Existing Shares) in Orphazyme A/S as a Result of an Issue of Bonus Shares to KLSDC and UCLB
The capital increase was decided pursuant to the Board of Directors’ authorization laid down in Article 3.6 of the Articles of Association.
Orphazyme: Publication of Manuscript Related to Phase II Trial Results for Arimoclomol in Patients With SOD1 ALS
The manuscript highlights arimoclomol’s favorable safety profile and provides data on efficacy that supports the continued development of arimoclomol in a Phase II/III trial.