FDA Advisory Committee to Review Arimoclomol for Niemann-Pick Disease Type C

On July 9, 2024, Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a company focused on rare disease therapeutics, announced that the U.S. Food and Drug Administration (FDA) will hold a meeting with the newly established Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024. The purpose of this meeting is to review the New Drug Application (NDA) for arimoclomol, an orally administered medication proposed as the first treatment of its kind for Niemann-Pick disease type C (NPC).

The FDA plans to release background materials for public access no later than two business days before the meeting. The GeMDAC is composed of specialists in medical genetics, inborn metabolic errors, epidemiology, and other relevant fields. Arimoclomol’s NDA has a Prescription Drug User Fee Act (PDUFA) action date scheduled for September 21, 2024.

Zevra's President and CEO, Neil F. McFarlane, expressed anticipation about presenting the NDA data to the GeMDAC. He emphasized that the meeting would provide an opportunity for both patients and members of the NPC community to share their experiences and underscore the urgent need for an approved treatment.

Niemann-Pick disease type C is a very rare, progressive, and neurodegenerative lysosomal storage disorder. It is characterized by the body's inability to transport cholesterol and other lipids within cells, leading to their buildup in various tissues, including the brain. This genetic disease stems from mutations in the NPC1 or NPC2 genes, which are crucial for producing lysosomal proteins. NPC can affect both children and adults, presenting a range of clinical symptoms. Individuals with NPC often lose their independence due to physical and cognitive impairments affecting speech, cognition, swallowing, movement, and fine motor skills. The disease progresses irreversibly and can be fatal within months, although it may take years for some individuals to be diagnosed and for the disease severity to increase.

Arimoclomol is Zevra’s investigational drug candidate for NPC and has received multiple designations from the FDA, including Orphan Drug, Fast Track, Breakthrough Therapy, and Rare Pediatric Disease designations. Additionally, the European Medicines Agency (EMA) has granted Orphan Medicinal Product designation for arimoclomol for the treatment of NPC. The FDA’s acceptance of the resubmitted NDA for arimoclomol and the assignment of a PDUFA action date marks a significant step in the path to making this treatment available to patients.

Zevra Therapeutics is dedicated to developing transformative therapies for rare diseases that have limited or no treatment options. The company's mission is to provide life-changing treatments to individuals affected by rare diseases. Utilizing data-driven development and commercialization strategies, Zevra is addressing complex challenges in drug development to bring new therapies to the rare disease community.

Zevra Therapeutics also offers expanded access programs, which are subject to its Expanded Access Program (EAP) policy. Participation in these programs is governed by the regulations of each jurisdiction in which the program operates and is determined at the discretion of the treating physician.