Marathon Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Deflazacort as a Potential Treatment of Duchenne Muscular Dystrophy

NORTHBROOK, Ill.--()--Marathon Pharmaceuticals, a biopharmaceutical company that develops new treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for deflazacort, a potential treatment for patients with Duchenne muscular dystrophy (DMD).

The rare pediatric disease designation supplements the Orphan Drug designation granted by the FDA in August 2013 for deflazacort in the treatment of patients with DMD. Marathon expects to submit a New Drug Application (NDA) for deflazacort, which also has Fast Track status, to the FDA in the first half of 2016.

The FDA defines a "rare pediatric disease" as a disease that affects fewer than 200,000 individuals in the U.S. primarily ages from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of an NDA or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for a subsequent marketing application for a different product.

DMD is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking and 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 development of deflazacort is part of a broader effort by Marathon to provide 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.

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.Based on published clinical studies, it appears that deflazacort may be an important treatment option for patients with DMD.4,5 The most common side effects reported in clinical trials 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.

About Duchenne Muscular Dystrophy (DMD)

DMD occurs as a result of mutations (mainly deletions) 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.


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. 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. Neuromuscul Disord 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.


Aileron Communications
Peter Gray, 312.629.9400

Release Summary

Marathon Pharmaceuticals announced that the FDA has granted rare pediatric disease designation for deflazacort, a potential treatment for patients with Duchenne muscular dystrophy (DMD).


Aileron Communications
Peter Gray, 312.629.9400