Fda Advisory Committee Votes Favorably That the Data Support Arimoclomol as Effective Treatment for Patients With Niemann-Pick Disease Type C

Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC). [...] “We are extremely pleased with the committee’s recognition of the benefits of arimoclomol for people living with NPC,” said Neil F. [...] “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, which consists of expert [...] The arimoclomol NDA has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024. [...] About Niemann-Pick Disease Type C (NPC): Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. [...] The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Dis [...] The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee goal action date (PDUFA date) of September 21, 2024. [...] About Zevra Therapeutics: Zevra Therapeutics is a rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug [...] Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the impact of meetings or communications with the FDA o [...] These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Zevra Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com Russo Partners Contacts Adanna G. Alexander, Ph. D.