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Zevra Rises on Update on Niemann-Pick Disease Type C Drug


Zevra Therapeutics, Inc. (NASDAQ: ZVRA) announced that the FDA has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (“GeMDAC”) to review the company’s new drug application (NDA) for pipeline candidate arimoclomol.

The NDA is seeking approval for arimoclomol, an orally delivered, first-in-class treatment for Niemann-Pick disease type C (“NPC”).

NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissues and organs, including the brain.

Investors were pleased with this positive regulatory update and shares of the company rose 21.94% on Jul 9 on the news.

The FDA will hold the meeting with GeMDAC on Aug 2 to review the NDA.

The regulatory body intends to make the background materials available to the public no later than two business days before the meeting.

The NDA for arimoclomol has been assigned a target action date of Sep 21, 2024.

Please note that the FDA extended the review period for the NDA in March 2024. The NDA, resubmitted in December 2023, …

Full story available on Benzinga.com

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