FDA Advisory Committee Supports Arimoclomol for Niemann-Pick Disease Type C

• The FDA's Genetic Metabolic Diseases Advisory Committee voted favorably (11-5) on the effectiveness of arimoclomol for treating Niemann-Pick Disease Type C (NPC).
• The committee's decision was based on pivotal trial data, long-term open-label extension study results, and expanded access programs, indicating arimoclomol's clinical benefit.
• Arimoclomol, developed by Zevra Therapeutics, has a PDUFA action date of September 21, 2024, for potential FDA approval as a treatment for NPC.
• NPC is an ultra-rare, progressive, neurodegenerative lysosomal storage disorder, and arimoclomol has received multiple designations, including Orphan Drug and Breakthrough Therapy designations.

Zevra Therapeutics' arimoclomol has received a favorable vote from the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) regarding its effectiveness in treating patients with Niemann-Pick disease type C (NPC). The committee voted 11-5 in favor of the drug, marking a significant step toward potential FDA approval. The Prescription Drug User Fee Act (PDUFA) action date for arimoclomol is set for September 21, 2024.

Clinical Data and Advisory Committee Review

The GeMDAC's positive recommendation was based on a comprehensive review of clinical data, including results from the pivotal trial, long-term data from an open-label extension study, and data from expanded access programs (EAP: NCT04316637). These data were recently presented at the 45th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD). The committee also considered feedback from independent experts, NPC patients, and patient advocacy groups.

Neil F. McFarlane, President and Chief Executive Officer of Zevra, expressed optimism about the continued path to approval, stating, "We are extremely pleased with the committee’s recognition of the benefits of arimoclomol for people living with NPC. Based on the totality of the clinical data...we remain confident in the clinical benefit offered by arimoclomol as a treatment for NPC."

Niemann-Pick Disease Type C (NPC)

NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by impaired intracellular cholesterol and lipid transport, leading to accumulation in various tissues, including the brain. The disease results from mutations in the NPC1 or NPC2 genes, affecting both children and adults with varying clinical presentations. Key neurological impairments include speech, cognition, swallowing, ambulation, and fine motor skills. The disease is irreversible and can be fatal.

Arimoclomol: A Potential Treatment for NPC

Arimoclomol is an orally-delivered investigational drug product candidate developed by Zevra Therapeutics for the treatment of NPC. It has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, as well as Orphan Medicinal Product designation by the European Medicines Agency (EMA).