MDA joins with the entire Duchenne muscular dystrophy community in celebrating the news of the U.S. Food and Drug Administration’s decision to grant approval for deflazacort (brand name Emflaza), under development by Marathon Pharmaceuticals, to treat the most common childhood form of muscular dystrophy.
“Today we celebrate FDA approval of Emflaza as a treatment option for kids and adults living with Duchenne muscular dystrophy,” said MDA President and CEO Steven M. Derks. “This approval follows decades of MDA research — including early-stage development and testing of Emflaza in DMD patients — that was made possible by the hard work, dedication and unwavering commitment of our donors and sponsors. Drugs like Emflaza aren’t developed in silos, but rather through the combined efforts of biotechs and drug companies, patient organizations like MDA, corporate partners and donors, and the families who are there every step of the way to see it through. We are grateful to be part of such a welcome new development in the DMD landscape — and, more importantly, to belong to such a giving and dedicated community.”
Emflaza, designed for use by people 5 years or older with Duchenne muscular dystrophy (DMD), regardless of genetic mutation, is the third drug in six months approved for diseases included in MDA’s program. It represents continued success for MDA and the entire DMD community.
Marathon Pharmaceuticals plans to make its commercial supply of Emflaza, in both tablet and oral suspension formulations, available as quickly as possible. An expanded access program (EAP) for the drug, called AccessDMD, will end soon, but Marathon has committed to ensuring continued product supply to patients while they transition to the commercial supply.
Emflaza, a corticosteroid, works as an anti-inflammatory and immunosuppressant. It has been shown to slow the loss of muscle strength and function, preserve cardiac and respiratory function, and reduce the incidence of curvature of the spine (scoliosis) in people with DMD. Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with Emflaza as compared to other steroids.
Corticosteroids are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of Emflaza for any use in the United States.
“Emflaza has shown meaningful benefits for patients living with DMD and has the potential to delay disease progression,” MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D., said. “With the FDA approval of Emflaza, we now have a treatment option for kids and adults with Duchenne, which is a major advance for the community. As a physician, I participated in a clinical trial of Emflaza and have a keen awareness about what the approval of this drug means for patients and families.”
MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including Emflaza, in DMD, and has invested more than $1.5 million in studies to determine drug effects, mechanism of action, side effects and best dosing regimen. To date, MDA has funded more than $200 million in DMD research.