FDA Grants Orphan Drug and Rare Pediatric Disease Status to Somite Therapeutics' SMT-M01 for Duchenne Muscular Dystrophy

Somite Therapeutics, a pioneering TechBio firm based in Boston, recently announced the U.S. Food and Drug Administration (FDA) has awarded both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to its leading program, SMT-M01, aimed at treating Duchenne muscular dystrophy (DMD).

Dr. Micha Breakstone, CEO and founder of Somite Therapeutics, expressed that obtaining these designations marks a crucial step for both the company and those affected by DMD. He stressed the importance of these recognitions in highlighting the unmet medical needs for treating DMD, as well as the potential of Somite's AI-driven solutions to create new cell therapies. Breakstone reaffirmed the company's commitment to advancing SMT-M01 rapidly through the clinical stages to provide a significant impact on the lives of DMD patients and their families.

The FDA’s Orphan Drug Designation is aimed at promoting the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. Meanwhile, the Rare Pediatric Disease Designation, along with the Priority Review Voucher Program, targets severe or life-threatening conditions primarily impacting children aged 0-18 and also affecting fewer than 200,000 individuals in the U.S. Upon approval, the Rare Pediatric Disease designation qualifies the product for a priority review voucher, which can expedite the marketing application for this or another product, or can be sold to another sponsor.

Dr. Kristy Brown, Senior Vice President of Translational Development at Somite Therapeutics, emphasized that these FDA designations not only validate the innovative potential of the SMT-M01 program but also highlight its capacity to address the critical unmet need in DMD treatment. Brown noted that the benefits accompanying these designations, such as tax credits for clinical trials, exemption from user fees, and the possibility of seven years of market exclusivity upon approval, are significant advantages as the company progresses with the clinical development of SMT-M01.

The SMT-M01 program is driven by Somite Therapeutics’ proprietary AlphaStem AI platform, which is focused on developing novel cell replacement therapies for DMD. Somite plans to initiate clinical trials for SMT-M01 within the next 18 months, with the objective of offering a mutation-agnostic treatment option for patients battling this debilitating disease.

Somite Therapeutics, founded in October 2023, is a venture-backed company that seeks to become a leader in stem cell biology, utilizing advanced AI models to develop human tissue for scalable cell therapies targeting diseases such as diabetes, obesity, and muscular dystrophies. The company's AlphaStem AI platform is designed to create a self-reinforcing cycle that enhances cell therapy development by generating extensive data, thereby accelerating the creation of therapies with wider applications. Somite's development pipeline includes the SMT-M01 program for Duchenne muscular dystrophy and the SMT-B01 program for metabolic disorders.

The company has secured over $10 million in funding to date. The executive team at Somite Therapeutics includes Micha Breakstone, PhD, as CEO and Co-founder, Jonathan Rosenfeld, PhD, as CTO and Co-founder, Carl Morris, PhD, as Chief Scientific Officer, Kristy Brown, PhD, as SVP of Translational Development, and Shai Kivity as VP of Strategy and Operations.

The scientific co-founders of Somite Therapeutics are prominent figures from esteemed institutions: Olivier Pourquie, PhD, Professor of Genetics and Pathology at Harvard Medical School and Brigham and Women’s Hospital; Allon Klein, PhD, Professor of Systems Biology at Harvard Medical School; Cliff Tabin, PhD, Chair of the Department of Genetics at Harvard Medical School; and Jay Shendure, PhD, HHMI Professor of Genome Sciences at the University of Washington.