FDA Grants Priority Review to Deflazacort for Duchenne Muscular Dystrophy
The Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to the New Drug Applications (NDA) of deflazacort immediate-release tablet and oral suspension formulations for the treatment of Duchenne Muscular Dystrophy (DMD).
The NDA filing was based on data from clinical studies evaluating deflazacort in patients with DMD, showing improved muscle strength, ambulation status, and other functional outcomes regardless of genetic etiology. In one of the pivotal, randomized, double-blind studies in 196 DMD patients, deflazacort demonstrated improved muscle strength compared to placebo at 12 weeks, and continued through the end of study at Week 52.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of February 2017 to make a decision on the NDA. If approved, deflazacort will be among the first commercially available treatments indicated for DMD in the U.S. In the meantime, Marathon has established Access DMD, an expanded access program (EAP) operating under FDA authorization, to make deflazacort available to U.S. patients at no cost. Additional information regarding the program can be found at www.AccessDMD.com or 1-844-800-4DMD (4363).
Deflazacort is an investigational glucocorticoid with anti-inflammatory and immunosuppressant properties.
For more information call (866) 562-4620 or visit Marathonpharma.com.