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Orphazyme A/S

http://www.orphazyme.com

Latest From Orphazyme A/S

When Breakthrough Falls Short: US FDA Complete Response Letters Rising Among BTD Applications For Approval

More than half of the complete response letters issued for applications holding breakthrough status have been issued since 2020, reflecting difficult – and often rancorous – efficacy debates.

Drug Review Review Pathway

Keeping Track: US FDA Issues Alzheimer’s Breakthrough Designations, Arimoclomol CRL, Rinvoq Delays

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.

US FDA Performance Tracker Approvals

Orphazyme Tumbles Back To Earth As FDA Rejects Arimoclomol

Following a week of "insane trading" which saw the Danish stock soar based on social media chatter, US regulators have surprised the firm and investors by issuing a complete response letter for arimoclomol for Niemann-Pick disease type C.

Complete Response Letters Rare Diseases

Orphazyme's Arimoclomol Disappoints For Muscle-Wasting Disorder

The Danish biotech's stock sank after arimoclomol failed in a Phase II/III trial for inclusion body myositis. Orphazyme is hoping for much better news in the coming weeks as the US FDA makes its decision on the drug as a treatment for Niemann-Pick disease Type C.

Rare Diseases Clinical Trials
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Company Information

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  • Pharmaceuticals
  • Biotechnology
    • Large Molecule
  • Other Names / Subsidiaries
    • Orphazyme US, Inc.
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