Usnoflast, a novel oral NLRP3 inhibitor, is being developed for the treatment of amyotrophic lateral sclerosis (ALS), a rare and progressive neurodegenerative disease. Usnoflast has been studied in several preclinical 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.

The USFDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States.

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, it added.

The company 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).

Pankaj Patel, chairman, Zydus Lifesciences, said, “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. Zydus is committed to unlocking new frontiers in neuroscience and developing Usnoflast for patients with ALS.”

Zydus Lifesciences is a discovery-driven, global life sciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies.