Marathon Pharmaceuticals, a biopharmaceutical company that develops new treatments for rare diseases, has announced that the FDA has granted rare pediatric disease designation for deflazacort, a potential treatment for patients with Duchenne muscular dystrophy (DMD).
The rare pediatric disease designation supplements the Orphan Drug designation granted by the FDA in August 2013 for deflazacort in the treatment of patients with DMD. Marathon expects to submit a New Drug Application (NDA) for deflazacort, which also has Fast Track status, to the FDA in the first half of 2016.
The FDA defines a "rare pediatric disease" as a disease that affects fewer than 200,000 individuals in the U.S. primarily ages from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of an NDA or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for a subsequent marketing application for a different product.
DMD is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking and breathing, and ultimately death. Diagnosis typically occurs between the ages of 2 and 5 with progressive weakness leading to a loss of ambulation in the pre-teen to teenage years. Though DMD affects approximately 15,000 boys in the United States, there are currently no approved therapies for the disease in the U.S.2
The development of deflazacort is part of a broader effort by Marathon to provide treatment options for patients with DMD and other rare diseases. Additionally, Marathon is developing a national precision medicine program in partnership with leading patient advocacy, research and commercial organizations to accelerate research on treatments for DMD. This effort will launch in the first quarter of 2016.