FDA Approves Aqneursa for Neurologic Symptoms of Niemann-Pick Disease Type C

By SPC Staff

The FDA approved levacetylleucine (Aqneursa, IntraBio) for treating neurologic symptoms of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg (33 lb). The approval comes days after the agency approved arimoclomol (Miplyffa, Zevra Therapeutics) for the same rare genetic disease.

NPC interferes with lipid transport within cells, causing progressive neurologic symptoms and organ dysfunction. Both levacetylleucine and arimoclomol (in combination