Zydus announces USFDA Orphan Drug Designation for Usnoflast in ALS treatment

Orphan drug designation offers development incentives and potential market exclusivity

Zydus has 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 designation provides eligibility for development incentives, including tax credits, user fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

Pankaj Patel, Chairman of Zydus Lifesciences Limited, commented, “This Orphan Drug Designation from the USFDA underlines the urgent need to develop Usnoflast to address ALS, a fatal neurodegenerative disease. Zydus is committed to unlocking new frontiers in neuroscience and develop Usnoflast for patients with ALS.”

ALS patients experience rapid neurodegeneration, leading to loss of movement, speech, and eventually respiratory failure.

The disease affects approximately 32,000 people in the USA, with around 5,000 new cases diagnosed annually. In Europe, over 30,000 people are estimated to live with ALS, while India has about 75,000 cases.

Usnoflast (ZYIL1) has been studied in various pre-clinical models, including Parkinson’s disease and Multiple Sclerosis. Zydus has previously completed a Phase 2(a) clinical trial in 24 ALS patients in India.

Zydus has also received approval from the USFDA to initiate a Phase 2(b) clinical trial for Usnoflast in ALS patients. This trial will be a randomized, double-blind, placebo-controlled study.

With the Orphan Drug Designation, Zydus aims to expedite the development of Usnoflast and provide a new treatment option for ALS patients.

The company’s commitment to addressing rare diseases is evident as it continues to push the boundaries of medical science.