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February 13, 2017

FDA approves Marathon's muscular dystrophy drug

  • Agency
  • Genetics/Genomics

WRAL TechWire – Marathon Pharmaceuticals, a U.S. research-based biopharmaceutical company focused developing of new treatments for rare diseases with manufacturing facilities in North Carolina, said the U.S. Food and Drug Administration (FDA) has 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 United States.

The company said the drug will be manufactured in tablet form by Alcami, a Wilmington-based contract development and manufacturing organization that also has a lab in Durham.

“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 United States that has never before been approved for the treatment of Duchenne muscular dystrophy anywhere in the world.