Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of patients with Duchenne muscular dystrophy (DMD), the most common and most severe form of muscular dystrophy. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.
“This NDA submission starts a process that we hope will result in broad access to this medication for all of those living with Duchenne who need it,” said Jeff Aronin, Chief Executive Officer, Marathon Pharmaceuticals. “We recognize the difficulty the Duchenne community has had in obtaining deflazacort and look forward to working closely with the FDA as they review our application.”
The NDA filing is supported by a full preclinical and clinical study program, including two pivotal clinical efficacy trials exclusively licensed by Marathon in more than 200 Duchenne patients 5 to 15 years of age. These data show 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 Marathon additionally conducted seven clinical pharmacology and safety studies of deflazacort and nine preclinical studies to support either the initiation of clinical studies or marketing approval. An expanded access program, Access DMD™, is ongoing in the United States and provides deflazacort to patients with Duchenne free of charge during the NDA review process.
Deflazacort is not currently approved in the United States for any indication. Versions of deflazacort are available in some countries outside the United States where it is approved for a number of indications, but not for Duchenne. The FDA has granted deflazacort Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for the treatment of Duchenne. If approved, deflazacort will be among the first commercially available treatments indicated for Duchenne in the United States. As of the date of Marathon’s FDA submission, there is no cure for Duchenne and currently no FDA-approved treatment.
“Duchenne muscular dystrophy is a devastating disease. Having broad access and clear guidance from the FDA on this treatment option, which has the potential to delay disease progression, would be an important step forward for our community,” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy. “We are committed to improving the care for people with this disease and are encouraged by this milestone and the critical efforts to persevere in the fight against Duchenne.”
Deflazacort, an investigational drug, is a glucocorticoid with anti-inflammatory and immunosuppressant properties.2 Based on data contained in the NDA and in published clinical studies, it appears that deflazacort may be an important treatment option for patients with Duchenne, if approved by the FDA. In one of the pivotal, randomized, double-blind, placebo controlled and active comparator studies that followed 196 patients with Duchenne, deflazacort met its primary endpoint of improved muscle strength versus placebo at 12 weeks.
Side effects that could occur with Deflazacort use include:
Weight gain, increased appetite, facial puffiness or Cushingoid appearance, unwanted hair growth, skin redness and headache.
Other less common but important side effects include: a decrease in the density of the bones (osteopenia) or fragility of the bones (osteoporosis) which may lead to fractures, acne, stomach upset or irritation to the stomach lining, cataracts (which can impair vision), increased susceptibility to infection, sugar intolerance and aggravation of diabetes, elevation in blood pressure, behavioral and mood changes, effects on growth and development such as short stature.
Deflazacort use is not recommended for patients who have a systemic fungal infection or are allergic to deflazacort or any of the inactive ingredients in deflazacort, have had recent or ongoing infections or have recently received a vaccine or are scheduled for a vaccination.
About ACCESS DMD™ (Marathon’s Expanded Access Program)
Marathon is currently making deflazacort available to qualified patients with Duchenne, at no cost, through ACCESS DMD™, an Expanded Access Program operating under FDA authorization. Patients, families and physicians can learn more about ACCESS DMD™, including a list of clinical sites participating in the program, by visiting www.AccessDMD.com or calling 1-844-800-4DMD (4363).
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is the most common and most severe form of muscular dystrophy.3 It affects mainly boys and young men, with an incidence of approximately 1 in 3,500 live male births.4 The disease is marked by progressive muscle weakening and wasting, leading ultimately to the inability to walk by the teen years or earlier, and severe respiratory and cardiac complications.5,6 Few patients live into their thirties.5
About Marathon Pharmaceuticals
Marathon Pharmaceuticals, LLC, is a biopharmaceutical company that develops treatments for rare diseases, with a focus on patients who currently have no treatment options. The company’s pipeline of new medicines includes 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 www.marathonpharma.com.