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- A panel of independent advisors to the U.S. FDA voted in favor of a marketing application filed by Zevra Therapeutics (NASDAQ:ZVRA) for its main active ingredient, arimoclomol, targeting Niemann-Pick disease type C (NPC), an ultra-rare disorder.
- In a meeting Experts on the FDA's advisory panel on genetic metabolic diseases voted 11 to 5 that the data behind arimoclomol demonstrate its efficacy against NPC, the company announced late Friday.
- Shares of Zevra (ZVRA) were in a mixed trading session Friday ahead of the meeting, which relates to the company's new drug application (NDA) for the oral therapy.
- FDA advisory committees issue nonbinding recommendations. However, the regulator usually follows them before deciding on approvals. In the case of arimoclomol, a final decision by the FDA is expected on or before September 21, 2024.
