Marathon Pharmaceuticals has announced its investigational medicine deflazacort will be accessible for free through an Expanded Access Program (ACCESS DMD) for patients with Duchenne muscular dystrophy (DMD).
Deflazacort will now be made available to eligible patients through a growing network of medical research centers across the nation. “In light of recent challenges faced by the DMD research community, we believe it is particularly important to step up as a company and get deflazacort into the hands of patients who may benefit, under the safeguards of a clinical trial protocol, while the company completes and FDA reviews the New Drug Application for marketing approval,” neurologist Jordan Dubow, Marathon’s CMO, said in a press release.
Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties. Previous clinical studies have shown the drug’s potential to become a new treatment option for DMD patients, with a great potential to slow down muscle degeneration, despite certain side effects.
Centers around the country, in cities like Little Rock, Arkansas, Houston, Texas, Hershey, Pennsylvania, and Seattle, Washington, participate in Marathon’s program, and more centers are expected to open.
“The Duchenne community desperately needs safe and effective treatment options, and Marathon’s ACCESS DMD program has the potential to make a real difference for our families, who do not have the luxury of time,” said Valerie A. Cwik, M.D., MDA’s executive vice president and CMO. “Expanded access to an important potential therapy option is a significant step forward as we work together as a community to help those with DMD live longer and grow stronger.”
Expanded access programs like ACCESS DMD enable patients who suffer from life-threatening diseases without approved treatments to receive an investigational drug for free, prior to full FDA approval, but with the agency’s evaluation and authorization.
DMD has no cure and has not been approved by the FDA. But deflazacort has shown promise, and the FDA has granted it Fast Track status, Orphan Drug designation, and Rare Pediatric Disease status. Marathon now plans to submit a New Drug Application for marketing approval to the FDA in May. If approved, the drug could become commercially available in the U.S. in January 2017.