The U.S. Food and Drug Administration (FDA) has accepted Marathon Pharmaceuticals’ New Drug Applications for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review.
Deflazacort, a glucocorticoid, works as an anti-inflammatory and immunosuppressant. In the United States, the drug is considered an investigational therapy, as it has not been approved by the FDA for any disorder. Versions of deflazacort are approved in other countries, but not for DMD.
FDA approval of the two NDAs, one for an immediate-release tablet-form of the experimental treatment and one for an oral suspension formulation, would allow widespread access to deflazacort for kids and adults with DMD across the United States and make the drug among the first FDA-approved treatments for Duchenne.
“The Duchenne community would greatly benefit from widespread and reliable access to a treatment option with the potential to delay disease progression,” said Valerie A. Cwik, M.D., MDA Executive Vice President and Chief Medical and Scientific Officer. “We are all too familiar with the challenges that children and adults with Duchenne and their families face, and we’re hopeful that an FDA approval of deflazacort would be one of the first of many treatments for Duchenne.”
In a pivotal study involving 196 participants with DMD, results showed that treatment with deflazacort improved muscle strength. Side effects reported to date for the drug include cushingoid appearance (puffiness and weight gain), abnormal hair growth, skin redness, swelling of the nasal passages or upper respiratory infection, irritability, and cataract formation.
Priority Review status is reserved for drugs that offer significant improvements over existing options or provide a treatment for a disorder for which no approved treatment currently exists, and shortens FDA review time from 10 months to a goal of six months. A decision on deflazacort, which previously has received Fast Track status, Orphan Drug designation and Rare Pediatric Disease status from the FDA, is anticipated in February 2017.
During the FDA review process, Marathon is making deflazacort available to people with Duchenne in the U.S., at no cost, through Access DMD, an expanded access program operating under FDA authorization. Patients, families and physicians can learn more about AccessDMD, including a list of clinical sites participating in the program, by visiting www.AccessDMD.com or calling (844) 800-4363.