Approximately 32,000 people in the USA are affected by ALS, with around 5,000 new diagnoses each year, while India estimates around 75,000 cases.

ALS typically has an average survival rate of two to five years after diagnosis, with most patients succumbing to respiratory failure due to rapid neurodegeneration.

Zydus Lifesciences, headquartered in Ahmedabad, Gujarat, India, is recognized as a global life sciences company engaged in the discovery, development, manufacturing, and marketing of a wide array of healthcare therapies.

The FDA's Office of Orphan Drug Products grants orphan status to facilitate the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S.

Zydus Lifesciences Ltd. announced on January 22, 2025, that its drug Usnoflast received orphan drug designation from the US Food and Drug Administration (FDA), a significant milestone for the company.

This designation has led to a rise in Zydus Lifesciences' stock price, which increased by 1.10% to Rs 987.60 following the announcement.

The company has received USFDA approval to start a Phase 2(b) clinical trial for Usnoflast in patients with Amyotrophic Lateral Sclerosis (ALS), which will be randomized and double-blind.

Previously, Zydus completed a Phase 2(a) trial involving 24 ALS patients in India, with plans to present the data at an upcoming medical conference and publish it in a medical journal.

The upcoming Phase 2(b) study will enroll 210 ALS patients to evaluate Usnoflast's efficacy and safety over a 36-week treatment phase, followed by a 16-week open label extension.

The primary endpoint of the trial will measure the change in ALSFRS-R total score, with key secondary endpoints including changes in Slow Vital Capacity and neurofilament levels in cerebrospinal fluid.

Usnoflast is an oral small molecule NLRP3 inflammasome inhibitor being developed for ALS, a fatal neurodegenerative disease that leads to muscle weakness and loss of voluntary control.

Pankaj Patel, Chairperson of Zydus Lifesciences, emphasized the urgent need for effective treatments for ALS, highlighting the challenges posed by the disease.