Orphazyme A/S announced today that arimoclomol has received US Fast Track designation in sporadic Inclusion Body Myositis.
Per the FDA, Fast Track is “a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.”
Together with Orphazyme, we are hopeful that this designation will expedite the FDA review of arimoclomol in sIBM should there be positive results in the phase 2/3 study. Results are expected in the first half of 2021.
What is Fast Track?
The Fast Track approval provided by the FDA allows Orphazyme to communicate more frequently during the review and approval process, and other potential benefits. Learn more about the FDA and Fast Track.
From the press release:
About Orphazyme A/S
Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins, including lysosomal storage diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.
Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood brain barrier, and has been studied in seven phase 1 and three phase 2 trials. Arimoclomol is in clinical development for NPC, Gaucher disease, sIBM, and ALS.
Sporadic Inclusion Body Myositis (sIBM) is a progressively debilitating muscle-wasting disease. sIBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability. The estimated prevalence of sIBM is 45.6 per million or 40,000 patients in the USA and Europe. There are no approved treatments for sIBM. Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of sIBM.