Duchenne MD Patients May Receive Investigational Drug at No Cost

by Margarida Azevedo, MSc | December 4, 2015

Marathon Pharmaceuticals has announced the launch of ACCESS DMD, its expanded access program (EAP) to provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) under the authorization of the U.S. Food and Drug Administration (FDA).

Marathon Pharmaceuticals, LLC, a biopharmaceutical company that develops treatments for rare neurological diseases, is preparing a pipeline of new treatments. Deflazacort, an anti-inflammatory and immunosuppressant glucocorticoid, is an investigational drug and not yet approved for use by the FDA. But DMD patients who enroll in ACCESS DMD, an open-label EAP, will receive deflazacort at no cost while the company continues to work toward its approval.

Expanded access programs open investigational drugs to patients with serious or immediately life-threatening diseases for which there are no comparable or alternative treatment options. Marathon expects to submit a New Drug Application for deflazacort for FDA review in the first half of 2016. If the application is successful, deflazacort could be commercially available by early 2017, the company said.

DMD is a recessive X-linked muscular dystrophy that leads to muscle degeneration, difficulties in walking and moving, and compromised pulmonary and cardiac function. To date, there is no cure for DMD and available therapies aim at minimizing symptoms to maintain the best possible quality of life.

Marathon works with a number of patient advocacy groups — such as Charley’s Fund, Coalition Duchenne, CureDuchenne, Foundation to Eradicate Duchenne, the Jett Foundation, Muscular Dystrophy Association, Parent Project Muscular Dystrophy and Ryan’s Quest — to spread the word about ACCESS DMD. “We have received great feedback on this program from the physicians and clinics that specialize in treating DMD and we are proud to launch it,” Jeff Aronin, CEO of Marathon Pharmaceuticals, said in a press release. “The ACCESS DMD Expanded Access Program is an important part of Marathon’s commitment to serving patients.”

More information about ACCESS DMD is available here, or by calling 1-844-800-4DMD (4363).

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About the Author

Margarida Azevedo, MSc Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.