A new drug by TechBio company Somite.ai for treating Duchenne Muscular Dystrophy has been granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the US Food and Drug Administration, Somite.ai said earlier this week.
Duchenne Muscular dystrophy (DMD) is a disease that causes progressive weakness and loss of muscle mass. Those afflicted are unable to carry out activities of daily living and must use wheelchairs. Affecting approximately two in every 10,000 born in the US, the National Library of Medicine has labeled DMD as one of the most severe forms of inherited muscular dystrophies.
Patients of DMD are unlikely to survive past the age of 30.
"Receiving both Orphan Drug and Rare Pediatric Disease Designations for SMT-M01 is a significant milestone for Somite.ai and, more importantly, for patients suffering from Duchenne muscular dystrophy," said Dr. Micha Breakstone, founder and CEO of Somite.ai.
"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 bring hope to DMD patients and their families," he added.
What does this mean?
An Orphan Drug can be defined as a drug that despite responding to a public health need, has not been developed by the pharmaceutical industry for economic reasons. The FDA provides orphan status to drugs and other biologics intended to prevent, diagnose, or treat rare diseases that affect fewer than 200,000 people in the United States.
Additionally, once approved, a Rare Pediatric Disease designation makes a product eligible for a priority review voucher, which can be used for marketing a separate product or even sold to another sponsor.
"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," Dr. Kristy Brown, SVP of Translational Development at Somite.ai said.
"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," she noted.
About Somite.ai
Somite.ai is a company that has developed AI foundation models to produce human tissue for cell therapies at scale for diseases such as diabetes, obesity, and muscular dystrophies. According to their site, their mission is to become the ‘OpenAI of stem cell biology.’