New Drug Applications (NDA) for Marathon’s investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA). The NDAs, one for immediate-release tablet formulations and one for an oral suspension formulation, request that deflazacort be approved for the treatment of Duchenne muscular dystrophy (DMD).
“This is an important milestone for Marathon but more importantly, for the Duchenne community,” Jeff Aronin, Marathon’s chief executive officer, said in a press release. “Today, a large number of those living with Duchenne who could potentially benefit from deflazacort do not get it simply because they do not have access. If deflazacort is approved, our goal is to work closely with the community and to make deflazacort widely available to Duchenne patients in the United States.”
Deflazacort is an investigational glucocorticoid with anti-inflammatory and immunosuppressant properties. The drug is not yet approved for any indication in the U.S. but a decision on the NDA is anticipated by February 2017. Deflazacort has already been granted Fast Track designation, Orphan Drug status and Rare Pediatric Disease status by the U.S. FDA.
Data attached to Marathon’s NDAs, published in clinical studies’ findings, suggests deflazacort may be a significant treatment option for DMD patients. In one pivotal, randomized, double-blind, placebo-controlled and active-comparator studies that evaluated deflazacort in 196 DMD patients, the investigational drug met its primary endpoint of improved muscle strength versus placebo at 12 weeks. Muscle strength continued to improve through the end of the study at week 52.
During the U.S. FDA review process, Marathon will make deflazacort available to American patients at no cost, through AccessDMD expanded access program. If approved, deflazacort will be among the first commercially available treatments for DMD in the U.S.
“The Duchenne community would greatly benefit from widespread and reliable access to a treatment option with the potential to delay disease progression,” said Dr. Valerie A. Cwik, Muscular Dystrophy Association’s 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.”