16 September 2020 - If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C.

Orphazyme today announced that the U.S. FDA has accepted, with priority review, the company’s new drug application for arimoclomol for the treatment of patients with Niemann-Pick disease Type C.

The FDA has set a target action date of 17 March  2021 under the Prescription Drug User Fee Act and has indicated that it does not currently plan to hold an advisory committee meeting to discuss the application.