• If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC)
• FDA has set target PDUFA action date of March 17, 2021

Copenhagen, Denmark, September 16, 2020 - Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today 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.

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 designation shortens the review period from the standard 10 months to six months from the acceptance of the NDA. The FDA has set a target action date of March 17, 2021 under the Prescription Drug User Fee Act (PDUFA) and has indicated that it does not currently plan to hold an advisory committee meeting to discuss the application.

Kim Stratton, Chief Executive Officer, Orphazyme, said, “The filing acceptance marks a significant milestone in our journey towards our first potential approval of arimoclomol for NPC, a devastating and often fatal disease for which there is no approved therapy in the U.S. We look forward to collaborating with the FDA as they complete their review of this NDA to address the unmet medical need in NPC, and meanwhile are working to expand our U.S. activities in preparation for potential commercial availability next year.”

“Acceptance of the arimoclomol filing by the FDA is another major step forward in the effort to bring a treatment to people affected by Niemann-Pick disease Type C and is a reflection of the commitment of the entire community coming together to support promising research,” said Joslyn Crowe, Executive Director of the National Niemann-Pick Disease Foundation.

Arimoclomol has been granted FDA Fast Track and Breakthrough Therapy Designations for NPC, as well as Orphan Drug and Rare Pediatric Disease Designations. Orphazyme expects to file a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA) for arimoclomol in NPC in H2 2020.