Marathon Pharmaceuticals, a U.S. research-based biopharmaceutical company focused solely on the development of new treatments for rare diseases, has announced that the FDA granted approval of EMFLAZA (deflazacort) for the treatment of Duchenne muscular dystrophy in patients 5 years and older. Duchenne, a severe form of muscular dystrophy, is a rare disease and fatal genetic disorder that affects about 15,000 people in the U.S.

“We are in a new era in the treatment of Duchenne muscular dystrophy. For the first time, patients in the U.S. with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition. We are pleased that this development will help patients with this disease stay stronger longer,” said Timothy M. Cunniff, Pharm.D., executive vice president, Research & Development, Marathon Pharmaceuticals. “This is only the first of our products to treat Duchenne muscular dystrophy and we are committed to serving this community of patients, researchers and advocates until a cure is found.”

The FDA granted EMFLAZA Priority Review, which is reserved for investigational medicines that may offer major advances in treatment over existing options. EMFLAZA is a new chemical entity in the U.S. that has never before been approved for the treatment of Duchenne muscular dystrophy anywhere in the world.

“We are pleased to learn that the FDA has approved EMFLAZA,” said Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the U.S. focused solely on Duchenne muscular dystrophy. “While steroids are considered Standard of Care for Duchenne patients, there has been no steroid specifically approved for Duchenne. The FDA approval of EMFLAZA provides options for our families when making crucial decisions about care with their providers. We hope that this approval gives more families access to this important medication."

Marathon Pharmaceuticals conducted 17 new preclinical and clinical studies and exclusively licensed two additional clinical studies to support the New Drug Application submissions to the FDA. This investment in research and development has led to new understanding of the dosing, drug interaction and safe use of EMFLAZA in the Duchenne patient population. Additional post-market research will continue to advance the science of the drug and the care of patients with Duchenne. One planned study will examine various dosage regimens in younger patients with Duchenne to determine if earlier intervention is safe and effective and ultimately impacts the course of the disease. A second planned study will examine various dosage regimens in non-ambulatory patients with Duchenne and characterize the pulmonary and cardiac effects of EMFLAZA.

“EMFLAZA is an important new drug with proven benefit in boys with Duchenne muscular dystrophy, increasing muscle strength and physical function, which is important for both patients and caregivers. In my own research and in the clinic deflazacort keeps boys walking longer. By undertaking the research needed to secure FDA approval of EMFLAZA, we now know more about the drug, its dosing and possible interactions. These are all advances in care for patients with Duchenne,” said Robert C. Griggs, M.D., University of Rochester Medical Center, a neurologist and an early investigator of deflazacort.

“My son James is living with Duchenne, and this important treatment option has had a positive effect on him. Today’s approval makes it possible for all patients living with Duchenne to get access to this new treatment option. That’s an important step forward. Marathon’s commitment to research—conducting the research needed for FDA approval and its ongoing clinical programs—means continued progress in the treatment of Duchenne,” said Joel Wood, father of a son with Duchenne and president of the Foundation to Eradicate Duchenne.