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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While Orphazyme's attempts to get approval for arimoclomol failed miserably, new owner KemPharm is confident it can get the oral heat shock protein amplifier across the regulatory finishing line for Niemann-Pick disease type C.