The US Food and Drug Administration (US FDA) has granted Orphan Drug Designation (ODD) to Zydus Lifesciences’ Usnoflast, an oral NLRP3 inhibitor, for treating amyotrophic lateral sclerosis (ALS). ALS is a rare neurodegenerative condition that affects motor neurons in the brain and spinal cord.
Zydus conducted a Phase 2(a) randomised, double-blind, placebo-controlled clinical trial for Usnoflast with 24 ALS patients across seven sites in India. The results of this study are expected to be presented at a medical conference and published in a journal. Following this, the US FDA has approved the initiation of a Phase 2(b) clinical trial for ALS patients.
Drugs receiving the ODD status benefit from several development incentives, such as tax credits for clinical testing, exemptions from certain user fees, and eligibility for 7 years of marketing exclusivity once approved by the USFDA.
ALS is a progressive and fatal disease that leads to muscle weakness, loss of voluntary movement, and eventual paralysis. Patients often lose their lives due to respiratory failure within two to five years of diagnosis.
Usnoflast has also been studied for other medical conditions, including Parkinson’s disease, inflammatory bowel disease (IBD), and multiple sclerosis (MS). Previously, the USFDA granted the drug ODD for treating Cryopyrin Associated Periodic Syndrome (CAPS), an autoinflammatory disease.
Zydus Lifesciences is a pharmaceutical company based in Ahmedabad, India. It focuses on developing treatments for unmet medical needs, including rare diseases. The company employs over 27,000 individuals globally and is involved in producing healthcare therapies.
Shares of Zydus Lifesciences Ltd, as of 11:46 AM, January 23, are trading at ₹987.25, up by 0.89% today, though it has declined by 14.07% over the past 6 months while gaining 35.40% over the past year.
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Published on: Jan 23, 2025, 2:29 PM IST
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