Zydus, a discovery-driven global pharmaceutical company, has announced that the United States Food and Drug Administration (USFDA) has granted Fast Track Designation to its investigational drug, Usnoflast, for the treatment of amyotrophic lateral sclerosis (ALS). This designation is intended to expedite the development and review of drugs that address serious conditions and fill an unmet medical need. It allows for more frequent communication with the FDA and makes the drug eligible for accelerated approval and priority review, provided certain criteria are met.
ALS is a progressive neurodegenerative disease that primarily affects motor neurons. Patients with ALS typically experience muscle weakness, loss of motor control, difficulty speaking and swallowing, and ultimately respiratory failure. The average life expectancy following diagnosis ranges from two to five years. Neuroinflammation and axonal degeneration are key features of the disease, contributing to the buildup of neurofilaments in cerebrospinal fluid and, eventually, in the bloodstream. This process is linked to the rapid progression and severe impact of ALS on physical function and quality of life.
Usnoflast, also known as ZYIL1, is an oral small molecule that targets the NLRP3 inflammasome pathway, which is believed to play a role in neuroinflammatory diseases. The drug has shown promise in pre-clinical studies involving models of neuroinflammation, Parkinson’s disease, Inflammatory Bowel Disease (IBD), and Multiple Sclerosis (MS). In India, Zydus completed a Phase 2(a) randomized, double-blind, placebo-controlled trial of Usnoflast in 24 ALS patients across seven clinical trial sites. The study is registered under the ClinicalTrials.gov Identifier NCT05981040.
Building on this research, Zydus has received USFDA approval to begin a Phase 2(b) clinical trial in the United States. This upcoming study will also be randomized, double-blind, and placebo-controlled, further evaluating the potential of Usnoflast as a treatment option for ALS. ALS affects an estimated 32,000 individuals in the U.S., with approximately 5,000 new cases diagnosed each year. In Europe, more than 30,000 people are believed to be living with the condition, and in India, the number is estimated to be around 75,000.
