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Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)
Date:3/9/2016

NORTHBROOK, Ill., March 9, 2016 /PRNewswire-USNewswire/ -- Marathon Pharmaceuticals, a leader in developing treatments for rare diseases, announced today that it is expanding patient access to deflazacort, its investigational medicine for Duchenne Muscular Dystrophy (DMD). Through Marathon's ACCESS DMD™ program, deflazacort is now being made available to qualified patients, at no cost, through a growing network of medical research centers across the country.   

"In light of recent challenges faced by the DMD research community, we believe it is particularly important to step up as a company and get deflazacort into the hands of patients who may benefit, under the safeguards of a clinical trial protocol, while the company completes and FDA reviews the New Drug Application for marketing approval," said Dr. Jordan Dubow, a board certified neurologist and Chief Medical Officer at Marathon.

Medical centers participating in ACCESS DMD™ are up and running across the country and the number is expected to increase rapidly. The medical centers currently approved are in Little Rock, AR; Chicago, IL; Kansas City, KS; Boston, MA; Charlotte, NC; Hershey, PA; Memphis, TN; Houston, TX; Norfolk, VA and Seattle, WA.

DMD is a rare disease affecting mainly young boys. It causes progressive muscle degeneration, and over time an inability to walk and conduct many daily activities. It also impairs pulmonary and cardiac function, and most patients do not live into their thirties.1 In clinical trials, deflazacort has shown the potential to slow muscle degeneration for patients with DMD.

"The Duchenne community desperately needs safe and effective treatment options and Marathon's ACCESS DMD™ program has the potential to make a real difference for our families, who do not have the luxury of time," said Valerie A. Cwik, M.D., Executive Vice President & Chief Medical and Scientific Officer of the Muscular Dystrophy Association. "Expanded access to an important potential therapy option is a significant step forward as we work together as a community to help those with DMD live longer and grow stronger."

There is no cure for DMD and no drug currently approved in the U.S. to treat this rare disease.  FDA has granted Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for deflazacort for the treatment of DMD. Marathon expects to submit a New Drug Application for deflazacort in May 2016 and, if approved by FDA, deflazacort could be made commercially available in the U.S. in January of 2017. Expanded access programs like ACCESS DMD™ allow patients suffering from a life threatening disease to receive an investigational drug, under FDA authorization, prior to full FDA evaluation.

Patients, families and physicians can learn more about ACCESS DMD™, including a list of clinical sites participating in the program, by visiting http://www.AccessDMD.com or calling 1-844-800-4DMD (4363).

Marathon is working with patient advocacy groups including Charley's Fund, Coalition Duchenne, CureDuchenne, Foundation to Eradicate Duchenne, the Jett Foundation, Michael's Cause, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, and Ryan's Quest to raise awareness of ACCESS DMD™.

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.

About Deflazacort
Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties.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., where it is approved for many uses, but not for DMD. Deflazacort is an investigational medication and is therefore not yet proven to be safe and effective for the treatment of patients with DMD.

About Duchenne Muscular Dystrophy
DMD occurs as a result of mutations in the dystrophin gene.1 These mutations lead to an absence of or defect in the protein dystrophin, which results in progressive muscle degeneration. The incidence is approximately 1 in 3,500 live male births.6 There is currently no cure for DMD.1,2,7 Treatment is generally aimed at controlling symptoms to maximize the quality of life.

References

  1. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93.
  2. Parent Project Muscular Dystrophy website. http://www.parentprojectmd.org/site/PageServer?pagename=CTCMD_cause. Accessed August 4, 2015.
  3. Wong BL, Christopher C. Corticosteroids in Duchenne muscular dystrophy: a reappraisal. Journal of Child Neurology 2002;17(3):183–9.
  4. Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle & Nerve 1994;17(4):386–91.
  5. Brooke MH. A randomised trial of deflazacort and prednisone in Duchenne muscular dystrophy: efficacy and toxicity. Neurology 1996;46:A476.
  6. Emery AE. Population frequencies of inherited neuromuscular diseases—a world survey. Neuromuscular Disorders 1991;1:19–29.
  7. Moxley RT, Ashwal S, Pandya S, et al. Practice parameter: Corticosteroid treatment of Duchenne dystrophy: Report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005;64:13-20.

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