Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA

NORTHBROOK, Ill.–(BUSINESS WIRE)–Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing
treatments for rare diseases, today announced it has submitted a New
Drug Application (NDA) to the U.S. Food and Drug Administration (FDA)
for the investigational drug deflazacort for the treatment of patients
with Duchenne muscular dystrophy (DMD), the most common and most severe
form of muscular dystrophy. The FDA has a 60-day filing review period to
determine whether the NDA is complete and acceptable for filing.
“This NDA submission starts a process that we hope will result in broad
access to this medication for all of those living with Duchenne who need
it,” said Jeff Aronin, Chief Executive Officer, Marathon
Pharmaceuticals. “We recognize the difficulty the Duchenne community has
had in obtaining deflazacort and look forward to working closely with
the FDA as they review our application.”
The NDA filing is supported by a full preclinical and clinical study
program, including two pivotal clinical efficacy trials exclusively
licensed by Marathon in more than 200 Duchenne patients 5 to 15 years of
age. These data show that deflazacort improved muscle strength and other
functional outcomes in patients with Duchenne regardless of genetic
etiology and in one of the studies ambulation status.¹
Marathon additionally conducted seven clinical pharmacology and safety
studies of deflazacort and nine preclinical studies to support either
the initiation of clinical studies or marketing approval. An expanded
access program, Access DMD™, is ongoing in the United States and
provides deflazacort to patients with Duchenne free of charge during the
NDA review process.
Deflazacort is not currently approved in the United States for any
indication. Versions of deflazacort are available in some countries
outside the United States where it is approved for a number of
indications, but not for Duchenne. The FDA has granted deflazacort Fast
Track status, Orphan Drug designation and Rare Pediatric Disease
designation for the treatment of Duchenne. If approved, deflazacort will
be among the first commercially available treatments indicated for
Duchenne in the United States. As of the date of Marathon’s FDA
submission, there is no cure for Duchenne and currently no FDA-approved
treatment.
“Duchenne muscular dystrophy is a devastating disease. Having broad
access and clear guidance from the FDA on this treatment option, which
has the potential to delay disease progression, would be an important
step forward for our community,” said Pat Furlong, Founding President
and CEO of Parent Project Muscular Dystrophy. “We are committed to
improving the care for people with this disease and are encouraged by
this milestone and the critical efforts to persevere in the fight
against Duchenne.”
About Deflazacort
Deflazacort, an investigational drug, is a glucocorticoid with
anti-inflammatory and immunosuppressant properties.² Based
on data contained in the NDA and in published clinical studies, it
appears that deflazacort may be an important treatment option for
patients with Duchenne, if approved by the FDA. In one of the pivotal,
randomized, double-blind, placebo controlled and active comparator
studies that followed 196 patients with Duchenne, deflazacort met its
primary endpoint of improved muscle strength versus placebo at 12 weeks.
Side effects that could occur with Deflazacort use include:

Weight gain, increased appetite, facial puffiness or Cushingoid
appearance, unwanted hair growth, skin redness and headache.

Other less common but important side effects include: a decrease in the
density of the bones (osteopenia) or fragility of the bones
(osteoporosis) which may lead to fractures, acne, stomach upset or
irritation to the stomach lining, cataracts (which can impair vision),
increased susceptibility to infection, sugar intolerance and aggravation
of diabetes, elevation in blood pressure, behavioral and mood changes,
effects on growth and development such as short stature.

Deflazacort use is not recommended for patients who have a systemic
fungal infection or are allergic to deflazacort or any of the inactive
ingredients in deflazacort, have had recent or ongoing infections or
have recently received a vaccine or are scheduled for a vaccination.
About ACCESS DMD™ (Marathon’s Expanded Access Program)
Marathon is currently making deflazacort available to qualified patients
with Duchenne, at no cost, through ACCESS DMD™, an Expanded Access
Program operating under FDA authorization. Patients, families and
physicians can learn more about ACCESS DMD™, including a list of
clinical sites participating in the program, by visiting www.AccessDMD.com
or calling 1-844-800-4DMD (4363).
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is the most common and most severe form of
muscular dystrophy.³ It affects mainly boys and young men,
with an incidence of approximately 1 in 3,500 live male births.⁴
The disease is marked by progressive muscle weakening and wasting,
leading ultimately to the inability to walk by the teen years or
earlier, and severe respiratory and cardiac complications.^5,6
Few patients live into their thirties.⁵
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.