MDA welcomes FDA approval for muscular dystrophy drug

MDA welcomes FDA approval for muscular dystrophy drug

The Muscular Dystrophy Association (MDA) has welcomed the U.S. Food & Drug Administration’s decision to approve Marathon Pharmaceuticals’ deflazacort drug to treat the most common childhood form of muscular dystrophy.

The deflazacort drug, labeled for use by individuals 5 years or older with Duchenne muscular dystrophy (DMD), can be used regardless of genetic mutation. It is third drug in 6 months approved by the FDA for diseases included in MDA’s program.

The approval marks a big step forward in the development of treatments for DMD and other related neuromuscular disorders, representing continued success for the entire DMD community.

MDA President Steven M. Derks said, “Today we celebrate FDA approval of Emflaza (deflazacort’s brand name) as a treatment option for kids and adults living with Duchenne muscular dystrophy … We are grateful to be part of such a welcome new development in the DMD landscape.”

Marathon said it would make its commercial supply of the deflazacort drug in both tablets and oral suspension formulations. AccessDMD, an expanded access program (EAP) for the new drug, is all set to end.

DMD is a muscle-wasting genetic disorder that primarily affects boys and cause problems with movement, heart and breathing. Individuals with the disorder usually lose the ability to walk, and typically die in their 20s.



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