Orphazyme announces U.S. FDA acceptance and Priority Review of New Drug Application for arimoclomol for Niemann-Pick disease Type C

Today, Orphazyme announced that the U.S. Food and Drug Administration (FDA) has accepted, with Priority Review, the company’s New Drug Application (NDA) for arimoclomol for the treatment of NPC disease. The FDA grants Priority Review to applications for potential therapies that, if approved, could offer a significant improvement in safety or effectiveness, diagnosis, or prevention of serious conditions.

This is a significant step and if approved, arimoclomol would become the first approved therapy in the U.S. for people with NPC disease.

The FDA has set target PDUFA action date of March 17, 2021.