Patients enrolled in ACCESS DMD™ will receive an investigational
drug, deflazacort, at no cost while participating in the program
NORTHBROOK, Ill.–(BUSINESS WIRE)–Marathon Pharmaceuticals, a biopharmaceutical company that develops
treatments for rare neurological diseases, today announced that, under
the authorization of the U.S. Food and Drug Administration (FDA), it
will provide deflazacort to qualified patients with Duchenne muscular
dystrophy (DMD) through an open-label, expanded access program (EAP)
DMD™. Deflazacort is an investigational therapy in the U.S. and has
not been approved by the FDA. Patients will be able to participate in
the ACCESS DMD™ expanded access program at no cost until deflazacort is
Expanded access programs provide a mechanism for early access to an
investigational drug to treat patients with a serious or immediately
life-threatening disease or condition that has no comparable or
satisfactory alternative treatment options.
DMD is a recessive X-linked form of muscular dystrophy, which results in
muscle degeneration, difficulty walking and loss of independent
ambulation, impaired pulmonary and cardiac function, and ultimately
death.1 There are currently no therapies approved in the U.S.
or other countries to treat DMD.
Marathon expects to submit a New Drug Application for deflazacort in the
first half of 2016, which 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.
Patients, families and physicians can learn more about ACCESS DMD™,
including a list of clinical sites participating in the program, by
or calling 1-844-800-4DMD (4363). Marathon has begun working with
patient advocacy groups including Charley’s Fund, Coalition Duchenne,
CureDuchenne, Foundation to Eradicate Duchenne, the Jett Foundation,
Muscular Dystrophy Association, Parent Project Muscular Dystrophy and
Ryan’s Quest to raise awareness of ACCESS DMD. Marathon will continue
its outreach in an effort to connect with all the patient groups who are
part of the DMD community.
“We have received great feedback on this program from the physicians and
clinics who specialize in treating DMD and we are proud to launch it
today,” said Jeff Aronin, CEO of Marathon Pharmaceuticals. “The ACCESS
DMD Expanded Access Program is an important part of Marathon’s
commitment to serving patients.”
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.
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, Orphan Drug designation and Rare
Pediatric Disease designation for deflazacort for the treatment of
patients with DMD.
About Duchenne Muscular Dystrophy
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.
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.
Parent Project Muscular Dystrophy website. http://www.parentprojectmd.org/site/PageServer?pagename=CTCMD_cause.
Accessed August 4, 2015.
Wong BL, Christopher C. Corticosteroids in Duchenne muscular
dystrophy: a reappraisal. Journal of Child Neurology 2002;17(3):183–9.
Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C.
Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle &
Brooke MH. A randomised trial of deflazacort and prednisone in
Duchenne muscular dystrophy: efficacy and toxicity. Neurology
Emery AE. Population frequencies of inherited neuromuscular diseases—a
world survey. Neuromuscul Disord 1991;1:19–29.
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
Peter Gray, 312-629-9400