AI-Powered Biotech Firm Somite.ai Secures Dual FDA Designations, Accelerating a Beacon of Hope for Families Facing This Devastating Genetic Disorder
Jerusalem Post Staff
September 18, 2024
What’s New
A breakthrough drug developed by Israel-based TechBio company Somite.ai for the treatment of Duchenne Muscular Dystrophy (DMD) has received simultaneous Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration. This achievement highlights Somite.ai’s commitment to leveraging AI-driven technology to combat severe genetic conditions.
Why It Matters
Duchenne Muscular Dystrophy is a debilitating disease marked by progressive muscle degeneration and weakness, ultimately leading to patients being unable to perform basic daily activities. Affecting roughly 2 in every 10,000 newborns in the U.S., it ranks among the most severe inherited muscular disorders. Tragically, most patients do not survive beyond 30 years of age. The dual FDA designations could accelerate new, effective treatments, offering a glimmer of hope to affected children and their families.
Key Quotes
“Receiving both Orphan Drug and Rare Pediatric Disease Designations for SMT-M01 represents a crucial 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 highlight the critical unmet need in DMD and the promise of our AI-driven approach to developing innovative cell therapies. We are dedicated to advancing SMT-M01 through clinical development as swiftly as possible to bring hope to DMD patients and their families.”
Dr. Kristy Brown, SVP of Translational Development at Somite.ai, elaborated, “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.”
Understanding the FDA Designations
An Orphan Drug receives its classification because it caters to a public health need that isn’t typically addressed by the pharmaceutical industry due to economic reasons. The FDA bestows orphan status on drugs aimed at preventing, diagnosing, or treating rare conditions affecting fewer than 200,000 people in the United States.
Moreover, a Rare Pediatric Disease designation comes with significant perks once approved, such as eligibility for a priority review voucher, which can expedite the marketing of another product or be sold to another company.
About Somite.ai
Somite.ai, a pioneering AI-driven biotech company, harnesses advanced AI foundation models to generate human tissue for scalable cell therapies targeting ailments like diabetes, obesity, and muscular dystrophies. Their ambition is to become the ‘OpenAI of stem cell biology.’
This story was first published on jpost.com.