Marathon Pharmaceuticals Submits NDA For the Treatment of Duchenne Muscular Dystrophy


Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of patients with Duchenne muscular dystrophy (DMD), the most common and most severe form of muscular dystrophy. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.

“This NDA submission starts a process that we hope will result in broad access to this medication for all of those living with Duchenne who need it,” said Jeff Aronin, Chief Executive Officer, Marathon Pharmaceuticals. “We recognize the difficulty the Duchenne community has had in obtaining deflazacort and look forward to working closely with the FDA as they review our application.”

The NDA filing is supported by a full preclinical and clinical study program, including two pivotal clinical efficacy trials exclusively licensed by Marathon in more than 200 Duchenne patients 5 to 15 years of age. These data show that deflazacort improved muscle strength and other functional outcomes in patients with Duchenne regardless of genetic etiology and in one of the studies ambulation status.1 Marathon additionally conducted seven clinical pharmacology and safety studies of deflazacort and nine preclinical studies to support either the initiation of clinical studies or marketing approval. An expanded access program, Access DMD™, is ongoing in the United States and provides deflazacort to patients with Duchenne free of charge during the NDA review process.

Deflazacort is not currently approved in the United States for any indication. Versions of deflazacort are available in some countries outside the United States where it is approved for a number of indications, but not for Duchenne. The FDA has granted deflazacort Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for the treatment of Duchenne. If approved, deflazacort will be among the first commercially available treatments indicated for Duchenne in the United States. As of the date of Marathon’s FDA submission, there is no cure for Duchenne and currently no FDA-approved treatment.

“Duchenne muscular dystrophy is a devastating disease. Having broad access and clear guidance from the FDA on this treatment option, which has the potential to delay disease progression, would be an important step forward for our community,” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy. “We are committed to improving the care for people with this disease and are encouraged by this milestone and the critical efforts to persevere in the fight against Duchenne.”

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