top of page

Zevra Therapeutics Media Release | March 4, 2024

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.


To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."

Ways to support NP-C in Australia:

34 views0 comments


Rated 0 out of 5 stars.
No ratings yet

Add a rating
bottom of page