Marathon Pharmaceuticals Receives FDA Fast Track Designation for Deflazacort as a Potential Treatment for Duchenne Muscular Dystrophy

Marathon Pharmaceuticals has announced that the FDA has granted Fast Track designation for deflazacort in the treatment of patients with Duchenne Muscular Dystrophy (DMD). DMD, a fatal disorder that causes the progressive deterioration of muscle fibers, is the most common and severe form of muscular dystrophy among children. Though DMD affects approximately 20,000 Americans, there are currently no approved therapies for the disease in the US.

FDA’s Fast Track programme is designed to facilitate the development of drugs that have demonstrated potential to treat diseases that are serious, life threatening, and for which there is an unmet medical need. Fast Track designation provides a number of benefits to facilitate drug development and approval, including the ability to meet and communicate more frequently with the agency to discuss drug development plans, as well as eligibility for Accelerated Approval and Priority Review of the New Drug Application (NDA).

Marathon is currently conducting numerous clinical and preclinical studies of deflazacort to support approval in patients with DMD. A NDA is anticipated in early 2016. FDA previously granted Orphan Drug designation to deflazacort for the treatment of DMD.

Marathon CEO, Jeffrey Aronin, remarked: “Marathon is committed to improving the lives of patients with Duchenne Muscular Dystrophy and other rare diseases. Fast Track designation for deflazacort is an important milestone for DMD patients and their families. We will work to take full advantage of this opportunity to accelerate availability to patients.”