The U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee voted in favor of the data supporting arimoclomol as an effective treatment for patients with Niemann-Pick disease type C (NPC).
NPC is a rare lysosomal storage disease characterized by variable clinical signs, depending on the age of onset. Common symptoms may include prolonged unexplained neonatal jaundice or cholestasis, isolated unexplained splenomegaly, and progressive, severe neurological symptoms such as cognitive decline, cerebellar ataxia, vertical supranuclear gaze palsy (VSPG), dysarthria, dysphagia, dystonia, seizures, gelastic cataplexy, and psychiatric disorders.
Clinical data from a pivotal trial, long-term data from an open-label extension study, and data from expanded access programs, as well as the committee’s recommendation, will all be reviewed by the FDA in hopes of an approval of arimoclomol. The treatment New Drug Application has a PDUFA action date of September 21, 2024.
Arimoclomal is an orally-administered first-in-class investigational drug that works to clear lipid build-up, improving lysosomal function.
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To learn more about rare lysosomal storage diseases, visit https://checkrare.com/diseases/lysosomal-storage-disorders/
Tags: genetic, metabolic, neuro, lung, lysosomal, regulations, drug development, treatment