FDA Panel Endorses Zevra's Arimoclomol for Niemann-Pick Disease

A panel of experts from the US Food and Drug Administration (FDA) has recommended that Zevra Therapeutics' arimoclomol be approved for treating Niemann-Pick disease type C (NPC). NPC is an extremely rare neurodegenerative lysosomal storage disorder, impacting approximately one in every 100,000 to 120,000 births.

The Genetic Metabolic Diseases Advisory Committee voted 11 to five in favor of the data submitted by Zevra, supporting arimoclomol as an effective treatment for NPC. The disease is characterized by mutations in the NPC1 or NPC2 genes, which result in an inability to properly metabolize lipids. This leads to a progressive loss of independence due to physical and cognitive limitations, affecting speech, cognition, swallowing, ambulation, and fine motor skills.

Arimoclomol is administered orally and works by increasing the production and activation of molecular chaperone proteins. These proteins help in breaking down the lipids that accumulate in the cells of individuals with NPC. The committee's recommendation was supported by long-term data from Zevra's expanded access program for arimoclomol, presented at the Inherited Metabolic Disorders Annual Meeting in April.

The data indicated that adults treated with arimoclomol, whether or not they were also receiving the substrate reduction therapy miglustat, generally maintained a stable disease course over two years of treatment and follow-up. Additionally, the safety profile of arimoclomol was consistent with observations from a phase 2/3 study.

Neil McFarlane, Zevra’s president and chief executive officer, expressed satisfaction with the committee's recognition of the benefits that arimoclomol offers to individuals living with NPC. He stated, “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, 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 FDA will now consider the committee's recommendation as it conducts its independent review of arimoclomol, with a final decision expected by 21 September. Arimoclomol has already received several designations from the FDA, including orphan drug, fast track, breakthrough therapy, and rare pediatric disease designations. Additionally, the European Medicines Agency has granted it orphan medicinal product designation.