Orphazyme Steams Ahead With Registration in Niemann Pick, Despite Endpoint Misses
Even with numerous endpoint misses, such is the demand for treatment options for the ultra-rare disease Niemann Pick, Orphazyme is pushing forward with its US and European regulatory filings of arimiclomol, which has no approved therapies in the US and one in the EU.
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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.
Finance Watch: Biopharma Stocks See Big Post-IPO Jumps
Public Company Edition: Five out of six drug developers and a CRO have seen their values spike immediately after going public this month. Also, Moderna floats a $500m offering, Revance proposes a $20m note sale, and Nestle invests another $200m in Aimmune.
Keeping Track: Submissions From Alkermes, Samsung Bioepis; BTDs for Orphazyme and Kiniksa
The latest submission and expedited review program news and highlights from our US FDA Performance Tracker.