RARE Daily

FDA Grants RPPD to Edgewise’s Muscular Dystrophy Program

November 30, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Edgewise Therapeutics’ EDG-5506 Orphan Drug designation for the treatment of Duchenne muscular dystrophy and Becker muscular dystrophy and Rare Pediatric Disease designation for the treatment of Duchenne.

EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker. EDG-5506 is currently advancing in multiple phase 2 trials for individuals with Duchenne, Becker, and other dystrophinopathies. The FDA previously granted Fast Track designation for the investigation and development of EDG-5506 for the treatment of Becker.

“Receiving Orphan Drug and Rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating individuals with Duchenne and Becker,” said Kevin Koch, president and CEO of Edgewise. “These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with these rare, serious or life-threatening disorders.”

The FDA grants orphan designation, also referred to as orphan status, to therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the United States. The designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval. The priority review voucher may be redeemed, transferred, or sold. Most recently, Sarepta Therapeutics sold a PRV for $102 million.

Duchenne muscular dystrophy is a severe, degenerative muscle disorder with a median life expectancy of around 30 years old. People living with Duchenne begin to lose their ability to walk without assistance by their early teens and nearly all will require the use of a wheelchair by the time they are in their mid-teens. Duchenne is the most common type of muscular dystrophy, and genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. Currently, there is no cure for Duchenne and therapeutic options are inadequate to prevent significant morbidity and mortality.

Becker is a genetic, progressive neuromuscular disorder that imposes significant physical, emotional, financial, and social impacts predominantly on males and their caregivers. Genetic mutations in the dystrophin gene resulting in Becker lead to contraction-induced muscle damage, which is the primary driver of muscle loss and impaired motor function in muscular dystrophies. Functional decline can begin at any age, and once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual’s life. Some individuals living with Becker experience heart failure from cardiomyopathy, which may result in heart transplantation or early death. Currently, there is no cure for Becker.

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