Marathon Pharmaceuticals announced this morning that the U.S. Food and Drug Administration (FDA) has accepted Marathon’s New Drug Applications (NDAs) for the investigational drug deflazacort for Priority Review. The NDAs are for an immediate-release tablet formulation and an oral suspension formulation of deflazacort for the treatment of patients with Duchenne muscular dystrophy.
The FDA granted deflazacort Priority Review status, which shortens the agency’s review time from 10 months to a goal of six months, meaning that the company anticipates a decision on the applications in February 2017. The FDA does not plan to hold an Advisory Committee meeting. More details are available in the press release, which is included below.
Marathon recognizes that the current process of accessing deflazacort is sometimes challenging and not available to everyone. If FDA approval is received, the company says its goal is to make deflazacort available and affordable to everyone who could benefit from this treatment and to work closely with the Duchenne community and clinicians on access and education.
As a reminder, deflazacort is currently available to qualified patients, at no cost, through ACCESS DMD™, an Expanded Access Program operating under FDA authorization. Information is available at www.AccessDMD.com or 1-844-800-4DMD (4363).
FDA Accepts Marathon Pharmaceuticals’ New Drug Applications for Deflazacort for the Treatment of Duchenne Muscular Dystrophy and Grants Priority Review
— FDA approval would allow widespread access for U.S. patients with Duchenne —
— Deflazacort could be among the first FDA-approved treatments for this devastating genetic disorder —
Northbrook, Ill. – August 10, 2016 – Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced the New Drug Applications (NDA) for the investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA). The NDAs (one for immediate-release tablet formulations and one for an oral suspension formulation) request approval for deflazacort for the treatment of patients with Duchenne muscular dystrophy (DMD), the most common and most severe form of muscular dystrophy.
The FDA’s Priority Review status is reserved for drugs that offer significant improvements over existing options or provide a treatment for a condition for which no approved treatment currently exists, and shortens the agency’s review time from 10 months to a goal of six months. A decision on the application is anticipated in February 2017 based on the Prescription Drug User Fee Act (PDUFA). Deflazacort previously received Fast Track status, Orphan Drug designation and Rare Pediatric Disease status from the FDA.
“This is an important milestone for Marathon but more importantly, for the Duchenne community,” said Jeff Aronin, Chief Executive Officer, Marathon Pharmaceuticals. “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. 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.”
The NDA filing included data from studies showing that deflazacort improved muscle strength and other functional outcomes in patients with Duchenne regardless of genetic etiology and in one of the studies ambulation status.1
“The Duchenne community would greatly benefit from widespread and reliable access to a treatment option with the potential to delay disease progression,” said Valerie A. Cwik, M.D., Executive Vice President and Chief Medical and Scientific Officer for the Muscular Dystrophy Association. “We are all too familiar with the challenges that children and adults with Duchenne and their families face, and we’re hopeful that an FDA approval of deflazacort would be one of the first of many treatments for Duchenne.”
During the FDA review process, Marathon is making deflazacort available to U.S. patients, at no cost, through Access DMDTM, an expanded access program (EAP) operating under FDA authorization. Patients, families and physicians can learn more about AccessDMD, including a list of clinical sites participating in the program, by visiting www.AccessDMD.com or calling 1-844800-4DMD (4363).