· Regulatory decisions expected in 2021
Copenhagen, Denmark, November 9, 2020 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for its investigational product candidate arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).
This follows Orphazyme’s U.S. NDA seeking approval for arimoclomol for the treatment of NPC that is under Priority Review with the FDA (PDUFA date: March 17, 2021). Arimoclomol has been granted Orphan Drug Designation in Europe and the U.S. Additionally, arimoclomol has FDA Fast Track and Breakthrough Therapy Designations for NPC as well as Rare Pediatric Disease Designations in the U.S.
Kim Stratton, Chief Executive Officer, said, “This filing in Europe is a significant milestone for Orphazyme as we work toward our first potential approvals of arimoclomol in major markets. There are few options today that can address the devastating effects of NPC, and we are hopeful we can address an important need for this community. We look forward to working with EMA as they complete their review of our application.”