Orphazyme Tumbles Back To Earth As FDA Rejects Arimoclomol
Drug Turned Down For NPC
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.
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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.
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.
Denmark’s Orphazyme has raised more funds to market its lead product, the heat shock protein amplifier, arimoclomol, in the US and Europe, and to evaluate the compound for other lysosomal storage and neurodegenerative disorders.