The Muscular Dystrophy Association said yesterday it welcomed news of the U.S. Food and Drug Administration’s decision to grant approval for deflazacort (Emflaza), developed by Marathon Pharmaceuticals, to treat the most common childhood form of muscular dystrophy.
Approval of the drug for Duchenne muscular dystrophy (DMD) patients 5 years or older is third drug in six months approved for diseases included in MDA’s program. The Association says it marks a solid step forward in the development of therapies for DMD and related neuromuscular diseases, and represents continued success for MDA and the entire DMD community.
“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.”