Zydus Lifesciences' Usnoflast Gets FDA's Orphan Drug Status

Zydus Lifesciences Ltd. said on Wednesday that the US Food and Drug Administration granted its underdevelopment drug Usnoflast the orphan drug designation, according to an exchange filing on Wednesday.

The FDA'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 2 lakh people in the United States.

Usnoflast is a new oral medicine that is being developed to treat amyotrophic lateral sclerosis. The disease affects nerve cells in the brain and spinal cord, leading to muscle weakness and loss of voluntary control over muscles. It is a fatal condition with an average survival of two–five years after diagnosis. Most patients die of respiratory failure, the disclosure added.

"This Orphan Drug Designation from the USFDA underlines the urgent need to develop Usnoflast to address Amyotrophic Lateral Sclerosis, which is a fatal neurodegenerative disease," Zydus Lifesciences Chairperson Pankaj Patel said.

He added that the company was committed to unlocking new frontiers in neuroscience and develop Usnoflast for patients with ALS.

The company also added that Usnoflast had been studied in several pre-clinical models of neuroinflammation, Parkinson's disease, inflammatory bowel disease and multiple sclerosis.

The FDA had earlier granted the drug an 'Orphan Drug Designation' to treat patients with Cryopyrin Associated Periodic Syndrome, a rare autoinflammatory disease.

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, the Ahmedabad-headquartered company stated.

It plans to present this Phase 2(a) trial data in an upcoming medical conference and publish in a medical journal.

Shares of Zydus Lifesciences closed 0.70% lower at Rs 978.85 apiece on the BSE, compared to a 0.75% advance in the benchmark Sensex.