December 1, 2023

FDA grants orphan status to Edgewise’s muscular dystrophy drug

BOULDER — The U.S. Food & Drug Administration has granted Orphan Drug Designation for a drug produced by Edgewise Therapeutics for the treatment of Duchenne and Becker muscular dystrophy.

Edgewise Therapeutics Inc. (Nasdaq: EWTX) is a muscle disease biopharmaceutical company. Its EDG-5506, in addition to Orphan Drug status, also received 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, the company said in a press statement. EDG-5506 is 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,” Kevin Koch, president and CEO of Edgewise, said in a written statement. “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.”


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The FDA grants ODD to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S. Potential benefits of the ODD include market exclusivity for the first ODD drug for an approved indication within the ODD for a seven-year period upon FDA approval, federal tax credit for qualified clinical research expenses incurred in the U.S., and a waiver of Prescription Drug User Fee Act fees (currently worth more than $4 million).

Rare Pediatric Disease Designation acknowledges therapies under investigation for rare pediatric diseases affecting fewer than 200,000 people in the U.S. with serious or life-threatening manifestations primarily affecting individuals up to 18 years of age. RPDD provides priority review of the marketing application, and if approved for marketing, grants that sponsor a priority review voucher which can be transferred or sold to another sponsor.

Duchenne is a severe, degenerative muscle disorder with a median life expectancy of about 30 years. 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. There is no cure for Duchenne.

Becker is a genetic, progressive neuromuscular disorder that imposes 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. There is no cure for Becker.In its most recent quarterly report, Edgewise reported higher expenses and losses as its drugs moved through the approval pipeline.

Lucas High
A Maryland native, Lucas has worked at news agencies from Wyoming to South Carolina before putting roots down in Colorado.
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