Copenhagen, Denmark, July 20, 2020 - Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response in order to develop and commercialize novel therapeutics for the treatment of neurodegenerative orphan diseases, today announced that it has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).

Arimoclomol has received FDA Fast-Track and Breakthrough Therapy Designations for NPC, as well as Orphan Drug and Rare Pediatric Disease Designations. The rolling NDA submission allowed Orphazyme to submit critical portions of its NDA to the FDA as they were completed. Following completion of submission, the FDA has up to 60 days to determine whether to accept the application for review.

“Data from the randomized, controlled, clinical trial of arimoclomol for Niemann-Pick disease Type C support the positive effect of this agent in stabilizing neurologic progression of the disease, specifically in subgroups of patients over four years of age, and in those also taking miglustat,” said Marc Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center in Rochester, MN. “The data show clear evidence of target engagement, specifically an elevation of Heat-Shock Protein levels, with encouraging changes in biomarkers of excess lipid storage. These data support the role of enhanced Heat-Shock Protein 70 expression in Niemann-Pick disease Type C and may have applications in other lysosomal disorders.”

“The NPC patient community’s need for disease-modifying therapy could not be more urgent as there are no FDA-approved treatments for this malicious disease,” said Justin Hopkin, MD, Board Chair of the National Niemann-Pick Disease Foundation (NNPDF). “On behalf of the NNPDF and the Niemann-Pick community, we would like to extend our heartfelt thanks to Orphazyme for their tireless commitment to improving the lives of NPC patients and congratulate them in their completed NDA submission for arimoclomol to the FDA as a treatment for NPC.”

Orphazyme expects to submit a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA) for arimoclomol in NPC in the second half of 2020.

Kim Stratton, Chief Executive Officer of Orphazyme, said, “The rapid completion of the rolling submission of arimoclomol in NPC brings us one step closer to the potential approval of a new treatment option that can address a substantial unmet need for patients with NPC. We look forward to working together with the FDA as they review our application. If approved, arimoclomol would be the only FDA-approved treatment for NPC in the U.S. This exciting step adds to the momentum at Orphazyme, as we make progress in our commercial preparations in the U.S. and other major markets ahead of potential approval in NPC, and as we progress arimoclomol through clinical trials in additional indications with unmet medical needs – Amyotrophic Lateral Sclerosis (ALS), sporadic Inclusion Body Myositis (sIBM), and Gaucher disease.”