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 US.
“This Orphan Drug Designation from the USFDA underlines the urgent need to develop Usnoflast to address Amyotrophic Lateral Sclerosis (ALS), which is a fatal neurodegenerative disease,” said Zydus’ chairman Pankaj Patel.
People living with ALS have an average survival of approximately two to five years from diagnosis, with most ALS patients dying from respiratory failure. ALS patients experience neuroinflammation and rapid neurodegeneration. ALS affects approximately 32,000 people in US and on an average 5,000 new patients are diagnosed every year with this disease in USA as per statistics from Centre for Disease Control and Prevention (CDC). More than 30,000 people are estimated to be living with ALS in Europe (European Union and United Kingdom), while India has an estimated 75,000 people living with ALS.
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 auto-inflammatory disease. Zydus has previously completed a Phase 2(a) randomized, double-blind, placebo controlled clinical trial in 24 ALS patients across 7 clinical trial sites in India. It is planningto present this Phase 2(a) trial data in upcoming medical conference and publish in medical journal. Zydus has recently received approval from USFDA to initiate a randomised, double blind, placebo- controlled Phase 2(b) clinical trial for Usnoflast in patients with ALS.
