Zydus Lifesciences Ltd. has announced that its experimental drug, Usnoflast, has been granted Fast Track Designation by the US Food and Drug Administration (US FDA) for the treatment of amyotrophic lateral sclerosis (ALS). This designation is given to drugs that treat serious conditions and fill an unmet medical need, providing benefits such as faster approval, priority review, and increased communication with the FDA. Usnoflast is an oral NLRP3 inhibitor designed to treat neuroinflammation associated with ALS, a debilitating and fatal neurodegenerative disease.
The Fast Track Designation comes after the US FDA granted Usnoflast Orphan Drug Designation (ODD), which provides incentives such as tax credits, fee access, and a seven-year market exclusivity period after marketing authorization. The ALS Association has welcomed the designation, highlighting the urgent need for effective treatments for ALS, a disease that is usually fatal within two to five years of symptom development.
Zydus has already conducted a Phase 2(a) trial with 24 ALS patients in India and has received US FDA approval to start a Phase 2(b) trial in ALS patients in the US. Pre-clinical studies have shown promise for Usnoflast in treating neuroinflammation, Parkinson’s disease, multiple sclerosis, and inflammatory bowel disease. ALS affects an estimated 32,000 Americans, with 5,000 new diagnoses each year, and over 30,000 people in Europe, with approximately 75,000 patients in India.
The Fast Track Designation and Orphan Drug Designation have enhanced Zydus’s position in the global neurodegenerative disease treatment sphere. The company is now poised to accelerate the development and review of Usnoflast, bringing hope to patients and families affected by ALS. With its clinical advancement and regulatory milestones, Zydus is making significant strides in addressing the unmet medical needs of patients with neurodegenerative diseases. The company’s efforts have the potential to improve the lives of thousands of people worldwide affected by these devastating diseases.