Marathon Pharmaceuticals announced the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for deflazacort for the treatment of patients with Duchenne Muscular Dystrophy (DMD)

The NDA submission was based on results from a preclinical and clinical program including two efficacy trials in more than 200 patients with DMD. Findings from these studies show that treatment with deflazacort improved muscle strength and other functional outcomes in DMD patients regardless of genetic etiology. Additional studies including seven clinical pharmacology and safety studies and nine preclinical trials were also conducted by the company. 

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The FDA previously granted deflazacort Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for the treatment of DMD.

Deflazacort is an investigational glucocorticoid with anti-inflammatory and immunosuppressant properties. If approved, it will be among the first commercially available treatments indicated for DMD in the US.

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