Deflazacort Wins Priority Review for Duchenne Muscular Dystrophy Drug

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Deflazacort is intended to help improve muscle strength and other functional outcomes in DMD regardless of genetic etiology.

Marathon Pharmaceuticals today announced that the US FDA has accepted its NDAs for its investigational drug deflazacort, indicated for the treatment of Duchenne muscular dystrophy (DMD).

The drug, available as an immediate release tablet and oral suspension, was also granted Priority Review, shortening the review period to 6 months. A decision is expected in February 2017.

“Today, a large number of those living with Duchenne who could potentially benefit from deflazacort do not get it simply because they do not have access,” Jeff Aronin, CEO of Marathon Pharmaceuticals, said in a statement. “If deflazacort is approved, our goal is to work closely with the community and to make deflazacort widely available to Duchenne patients in the United States.”

News of the NDA acceptance is just the latest in what has become a saga in the DMD space. Several attempts towards approval by other drug companies, including BioMarin and Sarepta, have been thwarted by the FDA as committee members struggle to justify drug approvals with lackluster clinical trial data.

In June, BioMarin announced that it would abandon further development of its DMD drugs after its investigational therapy drisapersen was officially denied by the FDA. Sarepta, meanwhile, has been stuck in limbo as the FDA has extended its review of its drug eteplirsen, for the treatment of DMD in patients with mutations amendable to exon 51 skipping, several times over the last few months.

Marathon’s deflazacort, which is intended to improve muscle strength and other functional outcomes in patients with DMD regardless of genetic etiology, met its primary endpoint of improved muscle strength at 12 weeks in a randomized, double-blind, placebo controlled and active comparator study in 196 patients with DMD. The improvements were sustained through the end of the study at 52 weeks.

During the review process, deflazacort will be available free of charge to patients in the US through the Access DMD™ expanded access program authorized by the FDA.

For more information about the expanded access program, go here.

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