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Emflaza

Generic name: deflazacort
Treatment for: Duchenne Muscular Dystrophy

Marathon Pharmaceuticals Receives FDA Fast Track Designation for Deflazacort as a Potential Treatment for Duchenne Muscular Dystrophy

Northbrook, Ill., January 19, 2015 – Marathon Pharmaceuticals, LLC, a biopharmaceutical company focused on developing treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for deflazacort in the treatment of patients with Duchenne Muscular Dystrophy (DMD). DMD, a fatal disorder that causes the progressive deterioration of muscle fibers, is the most common and severe form of muscular dystrophy among children. Though DMD affects approximately 20,000 Americans, there are currently no approved therapies for the disease in the U.S.

FDA’s Fast Track program is designed to facilitate the development of drugs that have demonstrated potential to treat diseases that are serious, life threatening, and for which there is an unmet medical need. Fast Track designation provides a number of benefits to facilitate drug development and approval, including the ability to meet and communicate more frequently with the agency to discuss drug development plans, as well as eligibility for Accelerated Approval and Priority Review of the New Drug Application (NDA).

Marathon is currently conducting numerous clinical and preclinical studies of deflazacort to support approval in patients with DMD. A NDA is anticipated in early 2016. FDA previously granted Orphan Drug designation to deflazacort for the treatment of DMD.

Marathon chief executive officer, Jeffrey Aronin, remarked: “Marathon is committed to improving the lives of patients with Duchenne Muscular Dystrophy and other rare diseases. Fast Track designation for deflazacort is an important milestone for DMD patients and their families. We will work to take full advantage of this opportunity to accelerate availability to patients.”

About Deflazacort

Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties.1 Based on published clinical studies, it appears that deflazacort may be an important new treatment option for patients with DMD.2,3 Side effects reported to date include cushingoid appearance, irritability and cataract formation.

About Duchenne Muscular Dystrophy (DMD)

DMD is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing, and death.4 The incidence is approximately 1 in 3,500 live male births.5 There is currently no cure for DMD.6,7 Treatment is generally aimed at controlling the onset of symptoms to maximize the quality of life.

References

  1. Wong BL, Christopher C. Corticosteroids in Duchenne muscular dystrophy: a reappraisal. Journal of Child Neurology 2002;17(3):183–9.
  2. 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.
  3. Brooke MH. A randomised trial of deflazacort and prednisone in Duchenne muscular dystrophy: efficacy and toxicity. Neurology 1996;46:A476.
  4. Yiu, E; Kornberg, A. “Duchenne muscular dystrophy”. Neurology India 2008;56(3):236-47.
  5. Emery AE. Population frequencies of inherited neuromuscular diseases—a world survey. Neuromuscul Disord 1991;1:19–29.
  6. 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.
  7. Bushby K, Muntoni F, Urtizberea A, et al. Report on the 124th ENMC International Workshop: Treatment of Duchenne muscular dystrophy: Defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord 2004;14:526-34.

Posted: January 2015

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