AdComm supports Zevra's genetic disorder drug

This week, the FDA's Genetic Metabolic Diseases Advisory Committee (GeMDAC) endorsed Zevra Therapeutics’ arimoclomol as a viable treatment for Niemann-Pick disease type C (NPC). The committee's decision was reached with 11 votes in favor and 5 against the orally-delivered heat shock protein amplifier. This determination was influenced by recent data presented at the Society for Inherited Metabolic Disorders meeting, supplemented by input from independent experts, NPC patients, and advocacy groups. The FDA will continue its independent evaluation of Zevra's New Drug Application (NDA) for arimoclomol, with a regulatory action date set for September 21, 2024.

Niemann-Pick disease type C is a rare, progressive neurodegenerative condition marked by the accumulation of cholesterol and lipids in various tissues, including the brain. This genetic disorder affects both children and adults, leading to significant physical and cognitive impairments. Patients with NPC experience symptoms such as difficulties with speech, cognition, swallowing, movement, and fine motor skills.

This is the second time arimoclomol has been presented to the FDA for approval. The agency initially rejected the drug in 2021 when it was owned by Denmark-based Orphazyme, citing a lack of sufficient qualitative and quantitative evidence of efficacy. Zevra, formerly known as KemPharm, acquired arimoclomol in May 2022, with the intention of refiling the NDA for NPC treatment.

In addressing the FDA's previous concerns, Zevra provided additional evidence supporting the use of the Niemann-Pick type C Clinical Severity Scale and conducted further studies to elucidate the drug's potential mechanism of action. The company's resubmission also included new trial data to bolster their case for arimoclomol's efficacy.

Niemann-Pick disease type C remains a challenging disorder with limited treatment options. The genetic condition not only disrupts cholesterol and lipid metabolism but also leads to severe neurological symptoms that significantly impact the quality of life for patients and their families. The GeMDAC's endorsement of arimoclomol represents a vital step forward in the ongoing effort to find effective treatments for this debilitating disease.

As the FDA continues its review process, the potential approval of arimoclomol offers hope to the NPC community. The collaborative effort between Zevra, independent experts, patients, and advocacy groups underscores the importance of comprehensive and patient-centered approaches in developing treatments for rare diseases. The forthcoming regulatory decision will be crucial in determining the future availability of arimoclomol as a treatment option for those suffering from NPC.