Arimoclomol fda review

The FDA's review of arimoclomol for the treatment of Niemann-Pick disease type C (NPC) has been a significant process with several key events:

1. **Initial Rejection and Resubmission**: In June 2021, the FDA issued a Complete Response Letter (CRL) to arimoclomol, stating that the drug would not be approved based on the data presented in the application. The CRL requested additional qualitative and quantitative evidence to support the validity of the 5-domain NPC Clinical Severity Scale (NPCCSS) used in the phase 2/3 trial. In response, Zevra Therapeutics resubmitted its NDA in December 2023 with additional evidence to address the FDA's concerns.
2. **FDA Advisory Committee Vote**: On August 2, 2024, the FDA's Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support arimoclomol as effective in treating patients with NPC. This recommendation will be considered by the FDA as it completes its independent review of the arimoclomol NDA.
3. **PDUFA Action Date**: The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024, for the arimoclomol NDA. This date is a target action date set by the FDA to complete its review of the application.
4. **Market Expectations**: The positive vote from the GeMDAC and the resubmission of the NDA have increased the probability of success of arimoclomol in NPC to 90% from 80%, as highlighted by analysts. The anticipated approval is seen as a significant opportunity for Zevra Therapeutics, given the lack of cure for NPC and the potential for arimoclomol to address this unmet medical need.

In conclusion, the FDA's review of arimoclomol has been a complex process with a recent positive advisory committee vote, reinforcing the drug's potential for approval. The company remains optimistic about the treatment's continued path to approval, with the final decision from the FDA expected on or before September 21, 2024.