Marathon Pharmaceuticals announced today that it is expanding patient access to deflazacort, its investigational therapy for Duchenne muscular dystrophy (DMD). Medical centers across the country are now participating in the open-label expanded access program (EAP) called ACCESS DMD to provide deflazacort to qualified U.S. patients at no charge.
Deflazacort, a corticosteroid, works as an anti-inflammatory and immunosuppressant. Based on published clinical studies, it appears that deflazacort may be an important new treatment option for patients with DMD. Side effects reported to date include cushingoid appearance, abnormal hair growth, weight gain, skin redness, nasopharyngitis, irritability, and cataract formation.
In the United States, deflazacort is considered an investigational therapy, as it has not been approved by the U.S. Food and Drug Administration (FDA).
MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including deflazacort, in DMD, with studies to determine the drug’s mechanism of action, drug effects, side effects and best dosing regimen.
To learn more about ACCESS DMD, including a list of clinical sites participating in the program, visit http://www.AccessDMD.com, or call 1 (844) 800-4363.
Expanded access programs provide a mechanism for early access to an investigational drug to treat patients with a serious or immediately life-threatening disease or condition that has no comparable or satisfactory alternative treatment options.
Marathon reported that it expects to submit a New Drug Application (NDA) for deflazacort in May 2016, which will be subject to review by the FDA. If approved, it says, deflazacort is expected to be made commercially available in the U.S. in January 2017.