Zevra Therapeutics Provides FDA Update On The PDUFA Action Date For Arimoclomol As A Treatment For Niemann-Pick Disease Type C
Read the full statement from Zevra Therapeutics here:
Here is a summary of the article:
Arimoclomol NDA Resubmission: Zevra resubmitted the New Drug Application (NDA) for arimoclomol, an investigational oral treatment for Niemann-Pick disease type C (NPC), a rare and fatal lysosomal storage disorder. The resubmission included additional analyses and data to support the efficacy and safety of arimoclomol. PDUFA Action Date Extension: The FDA extended the review period for the NDA and set a new Prescription Drug User Fee Act (PDUFA) action date of September 21, 20241. The FDA also confirmed its intention to hold an advisory committee meeting to discuss the resubmission.
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