FDA Advisory Committee Backs Arimoclomol As Effective Treatment For Niemann-Pick Disease Type C

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a company specializing in rare disease treatments, announced today that the Genetic Metabolic Diseases Advisory Committee (GeMDAC) of the U.S. FDA has voted in favor (11 yes, 5 no) of the effectiveness of arimoclomol for treating Niemann-Pick disease type C (NPC).

Neil F. McFarlane, President & Chief Executive Officer of Zevra, “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, including data from the pivotal trial, the long-term data from the arimoclomol open label extension study, and data from our expanded access programs (EAP: NCT04316637), we remain confident in the clinical benefit offered by arimoclomol as a treatment for NPC, and are optimistic about its continued path to approval.”

The GeMDAC, comprising experts in medical genetics, inborn errors of metabolism, epidemiology, and other specialties, evaluated the advantages and potential drawbacks of arimoclomol. Their discussion included data presented at the 45th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), along with feedback from independent experts, Niemann-Pick Disease Type C (NPC) patients, and representatives of patient advocacy groups. While the committee’s recommendation will inform the FDA’s ongoing review of the arimoclomol New Drug Application (NDA), it is not binding on the Agency. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024, for the arimoclomol NDA.

Niemann-Pick disease type C (NPC) is an extremely rare and progressive neurodegenerative disorder, categorized as a lysosomal storage disease. It results from the body's inability to transport cholesterol and other lipids within cells, causing these substances to accumulate in tissues, particularly in the brain.

The disorder is linked to mutations in the NPC1 or NPC2 genes, which produce lysosomal proteins. NPC affects both children and adults, presenting a range of symptoms. Those affected by NPC experience a gradual loss of independence due to physical and cognitive impairments, including difficulties with speech, cognition, swallowing, mobility, and fine motor skills. The progression of NPC is relentless and irreversible, often leading to a fatal outcome within months or taking years for diagnosis and worsening.