Orphazyme update – FDA reviews and does not approve the NDA for Arimoclomol.

Below, please find the official statement to the Niemann-Pick Type C community about the announcement that the U.S. Food and Drug Administration (FDA), following its review of the new drug application for arimoclomol for the treatment of NPC disease, will not be approved in its current form.

We are deeply saddened by this announcement and will work with Orphazyme and the patient community to find a path forward and keep access of Arimoclomol for families.