Zydus, a leading, discovery-based, global pharmaceutical company announced that the USFDA has granted ‘Fast Track Designation’ to Usnoflast, a novel oral NLRP3 inhibitor, for the treatment of amyotrophic lateral sclerosis (ALS). A drug that receives Fast Track Designation from the USFDA is eligible for accelerated approval and priority review, if the relevant criteria are met. The purpose of Fast Track Designation is to get important new drugs to the patients faster.

Speaking on the development, Chairman of Zydus Lifesciences Limited, Pankaj Patel, said, “This ‘Fast Track Designation’ in addition to the previous ‘Orphan Drug Designation’ granted by the USFDA underlines the urgent need to develop treatments to address Amyotrophic Lateral Sclerosis (ALS), which is a fatal neurodegenerative disease. Zydus is committed to unlocking new frontiers in neuroscience and develop Usnoflast for patients with ALS.”

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. Axonal neurodegeneration leads to formation of neurofilaments which first accumulate in CSF of ALS patients, and then slowly these neurofilaments enter blood circulation. Owing to rapid neurodegeneration, steady loss of the ability to move, speak, eat, eventually breathe, paralysis and death have been reported in ALS patients. ALS affects approximately 32,000 people in the U.S.A 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). 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. [ClinicalTrials.gov Identifier: NCT05981040]. 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 Amyotrophic Lateral Sclerosis (ALS).