Marathon Pharmaceuticals Begins New Drug Application Process for Deflazacort to Treat Duchenne Muscular Dystrophy

Following a pre-NDA meeting with the U.S. Food and Drug Administration (FDA), Marathon Pharmaceuticals has announced that it will begin the New Drug Application (NDA) process for deflazacort as a treatment for Duchenne muscular dystrophy (DMD).

Marathon expects to submit the NDA in the first quarter of 2016. If the application is approved, the company could make the drug commercially available in the United States in the first quarter of 2017.

Deflazacort, a corticosteroid, works as an anti-inflammatory and immunosuppressant. It has been shown to slow the loss of muscle strength and function, preserve cardiac and respiratory function, and reduce the incidence of scoliosis (curvature of the spine) in people with DMD. Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with deflazacort as compared to other steroids.

MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including deflazacort, in DMD, with studies to determine the drug’s mechanism of action, drug effects, side effects and best dosing regimen.

Deflazacort is available to DMD patients in the EU and Canada. The filing of an NDA by Marathon is an important step toward bringing this therapy to patients in the U.S.

See below to read Marathon Pharmaceuticals’ press release:

Marathon Pharmaceuticals to Begin New Drug Application Process for Deflazacort as a Potential Treatment for Patients with Duchenne Muscular Dystrophy

Northbrook, Ill. – August 05, 2015 – Marathon Pharmaceuticals, a biopharmaceutical company that develops new treatments for rare diseases, today 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).

DMD is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing and ultimately death.1 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 U.S.2

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.

Patients, families and physicians can receive notifications of Marathon’s clinical trials in DMD by visiting

The development of deflazacort is part of a broader effort by Marathon to provide new treatment options for patients with DMD and other rare diseases. Additionally, Marathon is developing a national precision medicine program in partnership with leading patient advocacy, research and commercial organizations to accelerate research on treatments for DMD. This effort will launch in the first quarter of 2016.

*investigational medications have not yet been approved by the FDA, and they have not been proven to be safe and effective.

About Marathon Pharmaceuticals
Marathon Pharmaceuticals, LLC, is a biopharmaceutical company that develops new treatments for rare diseases with a focus on providing medicine to patients who currently have no treatment options. The company is developing a pipeline of treatments for rare neurological and movement disorders. Marathon is headquartered in Northbrook, Illinois, with offices in Chicago, New Jersey and Washington D.C. For more information, visit

About Deflazacort
Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties.3 Based on published clinical studies, it appears that deflazacort may be an important new treatment option for patients with DMD.4,5  Side effects reported to date include cushingoid appearance, hirsutism, weight gain, erythema, nasopharyngitis, irritability and cataract formation.

Deflazacort is currently not approved in the U.S. but is available outside the U.S. for many approved uses not including DMD.  The FDA previously granted Fast Track status and Orphan Drug designation for deflazacort for the treatment of patients with DMD.

Roxan Olivas
MDA Vice President  — Public Relations & Community Programs
(520) 529-5317