This week, the FDA's recently-formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of recognizing Zevra Therapeutics’ arimoclomol as an effective treatment for Niemann-Pick disease type C (NPC).
The committee's decision, with 11 votes in favor and 5 against for the orally-delivered heat shock protein amplifier, was supported by recent data from the Society for Inherited Metabolic Disorders meeting and considered input from independent experts, NPC patients and advocacy groups. The FDA will continue its independent review of Zevra's NDA for arimoclomol, which has a regulatory action date of September 21, 2024.
Niemann-Pick disease type C is a rare and progressive neurodegenerative disorder characterized by the accumulation of cholesterol and lipids in various tissues, including the brain. The genetic condition affects both children and adults, leading to severe physical and cognitive impairments. Symptoms include difficulties with speech, cognition, swallowing, movement and fine motor skills.
This is arimoclomol's second attempt with the FDA. In 2021, the agency rejected the drug, then owned by Denmark-based Orphazyme, citing the need for additional qualitative and quantitative efficacy evidence. Zevra, formerly known as KemPharm, acquired the drug in May 2022 with plans to refile the NDA for arimoclomol in NPC.
Zevra addressed the FDA's previous concerns by providing additional evidence to support the use of the Niemann-Pick type C Clinical Severity Scale and conducting further studies to clarify the potential mechanism of action. The company also included new trial data in the resubmission.
