Zydus Lifesciences announced that the USFDA has granted Orphan Drug Designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for the treatment of amyotrophic lateral sclerosis (ALS).
The USFDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States.
Usnoflast (ZYIL1) is a novel, oral small molecule NLRP3 inhibitor. Usnoflast has been studied in several pre-clinical models of neuroinflammation, Parkinson’s disease, Inflammatory Bowel Disease (IBD) and Multiple Sclerosis (MS). The USFDA has earlier granted Zydus an ‘Orphan Drug Designation’ for Usnoflast to treat patients with Cryopyrin Associated Periodic Syndrome (CAPS), a rare autoinflammatory disease.
Zydus has previously completed a Phase 2(a) randomised, double-blind, placebo-controlled clinical trial in 24 ALS patients across seven clinical trial sites in India. It is planned to present this Phase 2(a) trial data in an upcoming medical conference and publish it in a medical journal.
Zydus has also recently received approval from the USFDA to initiate a randomised, double-blind, placebo-controlled Phase 2(b) clinical trial for Usnoflast in patients with Amyotrophic Lateral Sclerosis (ALS).