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Orphazyme A/S
Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases.
We are harnessing amplification of Heat-Shock Proteins (or HSP’s) in order to develop and commercialize novel therapeutics for serious diseases including lysosomal storage diseases and neuromuscular degenerative diseases.
Heat-Shock proteins (HSPs) are a group of endogenous stress-response proteins which protect against cellular toxicity caused by misfolded proteins, protein aggregation and lysosomal dysfunction. Our product candidate, arimoclomol, is designed to selectively amplify HSP’s.
The U.S. Food and Drug Administration (FDA) has accepted, with priority review, our New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC) and submitted a Marketing Authorisation Application to the European Medicines Agency (EMA) for NPC in November 2020.
We also have registrational trials ongoing in Amyotrophic Lateral Sclerosis (ALS) and sporadic Inclusion Body Myositis (sIBM), and intend to advance arimoclomol into pivotal-stage clinical development in neurological Gaucher disease.
