FDA accepts New Drug Application (NDA) for arimoclomol for treatment of Niemann-Pick disease Type C (NPC)

NPC is rare, genetic lysosomal storage disease characterised by accumulation of lipids in tissues and organs. Arimoclomol amplifies production of heat-shock proteins, which can rescue defective misfolded proteins, clear protein aggregates, and improve lysosome function.


Biospace Inc.