Following the successful trial, a new drug application (NDA) has been submitted to the Food and Drug Administration (FDA).
The study, sponsored by IntraBio, evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann–Pick disease type C. The trial found that, among patients with Niemann–Pick disease type C, treatment with NALL for 12 weeks led to better neurologic status than placebo. A longer period is needed to determine the long-term effects of this agent in patients with Niemann–Pick disease type C.
IntraBio is a spin out from Pharmacology co-founded by Antony Galione, Grant Churchill and Fran Platt. For the full paper, see https://www.nejm.org/doi/full/10.1056/NEJMoa2310151