FDA Advisory Committee to Review Arimoclomol for Niemann-Pick Disease Type C on August 2, 2024

CELEBRATION, Fla., July 09, 2024 – Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a company focused on rare disease treatments, has announced that the U.S. Food and Drug Administration (FDA) will hold a meeting on August 2, 2024, with the newly established Genetic Metabolic Diseases Advisory Committee (GeMDAC). The committee will review the New Drug Application (NDA) for arimoclomol, an innovative oral treatment for Niemann-Pick disease type C (NPC).

The FDA has stated that all relevant background materials for the meeting will be made publicly accessible at least two business days prior. The GeMDAC is composed of specialists in medical genetics, metabolic disorders, epidemiology, and related areas. The NDA for arimoclomol has a Prescription Drug User Fee Act (PDUFA) action date set for September 21, 2024.

Neil F. McFarlane, President and CEO of Zevra, expressed anticipation for presenting the arimoclomol data to GeMDAC. He emphasized that the meeting would also allow patients and the NPC community to share their experiences and underscore the urgent need for approved treatments.

Niemann-Pick disease type C (NPC) is an ultra-rare, progressive disorder that affects lysosomal storage, impairing the body's ability to transport cholesterol and other lipids within cells. This leads to excessive accumulation of these substances in tissues and organs, including the brain. NPC is caused by mutations in the NPC1 or NPC2 genes, which are essential for producing lysosomal proteins. The disease affects both children and adults, presenting a range of clinical symptoms. Those living with NPC experience physical and cognitive limitations, with key neurological impairments in speech, cognition, swallowing, movement, and fine motor skills. The progression of NPC is irreversible and can be fatal, with the severity and timeline of the disease varying widely.

Arimoclomol is Zevra’s first-in-class investigational drug for treating NPC. It has received several designations from the FDA, including Orphan Drug, Fast Track, Breakthrough Therapy, and Rare Pediatric Disease designations. The European Medicines Agency (EMA) has also granted it Orphan Medicinal Product designation for NPC treatment. The FDA's acceptance of the resubmitted NDA for arimoclomol sets a PDUFA date of September 21, 2024.

Zevra Therapeutics is dedicated to developing treatments for rare diseases, combining scientific research, data analysis, and patient needs to create significant therapeutic advancements. The company's mission is to deliver life-changing treatments for those with limited or no current options, employing unique, data-driven strategies to navigate the complexities of drug development.

Zevra also offers expanded access programs, subject to the company’s policies and relevant regulations, allowing patients to access investigational drugs outside of clinical trials. Eligibility for participation in these programs is determined by the treating physician based on specific criteria. 

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