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news.Marathon Pharmaceuticals, a biopharmaceutical company that develops new treatments for rare diseases, announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients with Duchenne muscular dystrophy (DMD) following a positive pre-NDA meeting with the U.S. Food and Drug Administration (FDA).
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DMD is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing and ultimately death.
Diagnosis typically occurs between the ages of 2 and 5 with progressive weakness leading to a loss of ambulation in the pre-teen to teenage years.
Though DMD affects approximately 15,000 boys in the United States, there are currently no approved therapies for the disease in the US.
The deflazacort NDA is expected to be submitted in the first quarter of 2016 and will be subject to review by the FDA. If approved, deflazacort is expected to be made commercially available in the U.S. in the first quarter of 2017. The FDA previously granted Fast Track status and Orphan Drug designation for deflazacort for the treatment of patients with DMD.
Marathon has requested authorization from the FDA to provide deflazacort as an investigational drug to qualified patients with DMD at no cost, through an expanded access program (EAP).
FDA indicated that it may formally authorize this program pending a full review of the EAP protocol. If the program is authorized by FDA, doctors will be able to enroll qualified patients to receive this investigational* medication beginning in September 2015.