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New drug approved for rare childhood disease

A new drug from TechBio company somite.ai to treat Duchenne muscular dystrophy has received both orphan drug (ODD) and rare pediatric disease (RPDD) designation from the U.S. Food and Drug Administration, somite.ai announced earlier this week.

Duchenne muscular dystrophy (DMD) is a disease that causes progressive weakness and loss of muscle mass. Sufferers are unable to perform activities of daily living and are dependent on wheelchairs. It affects approximately two in 10,000 people in the United States. The National Library of Medicine has classified DMD as one of the most severe forms of inherited muscular dystrophies.

Patients with DMD are unlikely to live beyond the age of 30.

“Receiving both orphan drug and rare pediatric disease designation for SMT-M01 is a significant milestone for Damite.ai and, more importantly, for patients suffering from Duchenne muscular dystrophy,” said Dr. Micha Breakstone, Founder and CEO of Damite.ai.

“These awards underscore the critical unmet need in DMD and the potential of our AI-driven approach to developing innovative cell therapies. We are committed to advancing SMT-M01 through clinical development as quickly as possible to provide hope to DMD patients and their families,” he added.

What does this mean?

An orphan drug can be defined as a drug that meets a public health need but was not developed by the pharmaceutical industry for economic reasons. The FDA grants 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.

Pills on a health form (Source: INGIMAGE)

In addition, once approved, the classification of a product as a rare childhood disease entitles the product to a Priority Review Voucher, which can be used to market a separate product or even sell it to another sponsor.

“These FDA approvals validate the innovative nature of our SMT-M01 program and its potential to address the significant unmet medical need in the treatment of Duchenne muscular dystrophy,” said Kristy Brown, Ph.D., SVP of Translational Development at Damite.ai.

“The designations will provide us with important benefits as we advance SMT-M01 through clinical development, including tax credits for qualified clinical trials, royalty exemptions, and eligibility for seven years of market exclusivity following regulatory approval,” she noted.

About somite.ai

Damite.ai is a company that has developed basic AI models to engineer human tissue at scale for cell therapies against diseases such as diabetes, obesity and muscular dystrophy. According to their website, their mission is to become the “OpenAI of stem cell biology.”



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