Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein (HSP) response for the treatment of neurodegenerative orphan diseases. We have clinical trial programs underway in four areas to explore the potential of our lead investigational treatment, arimoclomol.
Heat shock proteins, such as heat shock protein 70 (HSP70), are a group of endogenous stress-response proteins that protect against cellular toxicity caused by misfolded proteins, protein aggregation, and lysosomal dysfunction. Our product candidate arimoclomol is designed to selectively amplify HSPs.
The U.S. Food and Drug Administration (FDA) has accepted our new drug application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC) and designated it for priority review. We submitted a marketing authorization application to the European Medicines Agency (EMA) for NPC in November 2020.
We also have a registrational trial ongoing in amyotrophic lateral sclerosis (ALS) and intend to advance arimoclomol into pivotal-stage clinical development in neurological Gaucher disease. On March 29, 2021, the Company reported topline results from the Phase 2/3 trial in IBM; arimoclomol did not meet the primary and secondary endpoints in the Phase 2/3 trial in IBM. Additional analyses are being performed and the Company expects to present further information at an upcoming scientific forum.
ARIMOCLOMOL: PIPELINE-IN-A-PRODUCT POTENTIAL
Scroll over the pipeline chart to read more about each disease area.
U.S. FDA accepted New Drug Application (NDA) for review; Submitted MAA (EU) 2020
Top-line results reported on March 29, 2021; did not meet primary and secondary endpoints in IBM
***Type 1 and Type 3 Gaucher disease.
Rare pediatric disease designation