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 an MAA with the EMA for arimoclomol in NPC in H2 2020.

by | Sep 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.

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Sten Verland, PhD MSc

General Partner