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Orphazyme A/S

http://www.orphazyme.com

Latest From Orphazyme A/S

Orphazyme Wants More Time To Deal With Late-Stage Issues For Arimoclomol In EU

Following Orphazyme’s disappointment in the US regarding its investigational drug for an ultrarare disease, the company has said it will need more time to address outstanding issues raised by EU reviewers about the same medicine.

Europe Drug Review

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
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Company Information

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