Marathon Pharmaceuticals, LLC To Begin New Drug Application Process For Deflazacort As A Potential Treatment For Patients With Duchenne Muscular Dystrophy

NORTHBROOK, Ill.--(BUSINESS WIRE)--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 http://www.AccessDMD.com.

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 www.marathonpharma.com.

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.

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.

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. 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.