Somite Therapeutics has announced that the US Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company's lead program, SMT-M01, for the treatment of Duchenne muscular dystrophy (DMD).

"Receiving both Orphan Drug and Rare Pediatric Disease Designations for SMT-M01 is a significant milestone for Somite Therapeutics and, more importantly, for patients suffering from Duchenne muscular dystrophy. These designations underscore the critical unmet need in DMD and the potential of our AI-driven approach to develop innovative cell therapies. We are committed to advancing SMT-M01 through clinical development as rapidly as possible to make a meaningful difference for DMD patients and their families," said Dr. Micha Breakstone, Founder and CEO of Somite Therapeutics.

The FDA's Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States.

Rare Pediatric Disease Designation and Priority Review Voucher Program are granted for serious or life-threatening diseases that primarily affect individuals aged from birth to 18 years and impact fewer than 200,000 people in the US.

Once approved, the RPD designation also makes the product eligible for a priority review voucher. This voucher can be used for a subsequent marketing application for a different product or sold to another sponsor to expedite their marketing application review.

"These FDA designations validate the innovative nature of our SMT-M01 program and its potential to address the significant unmet medical need in Duchenne muscular dystrophy. The designations will provide important benefits as we advance SMT-M01 through clinical development, including tax credits for qualified clinical trials, exemption from user fees, and eligibility for seven years of market exclusivity upon regulatory approval," commented Dr. Kristy Brown, SVP Translational Development at Somite Therapeutics.

Somite Therapeutics' SMT-M01 program leverages the company's proprietary AlphaStem AI platform to develop a novel cell replacement therapy for DMD. The company plans to initiate clinical trials for SMT-M01 in the next 18 months, with the goal of providing a mutation-agnostic therapeutic for patients with this devastating disease.