ACCESS DMD™: An open-label, expanded access protocol intended to provide treatment with
MP-104 (deflazacort)* to U.S. children, adolescents, and/or adults with Duchenne muscular
dystrophy
**may include program/institution/advocacy, etc. logo
[INSERT SITE CONTACT INFORMATION]
[Date]
Dear Physician,
I am writing to share information with you about an expanded access program for patients with Duchenne
muscular dystrophy (DMD) called ACCESS DMD™. The intent of an expanded access program is to
provide patients with access to investigational medication for serious diseases or conditions where there
is no comparable or satisfactory alternative therapy available. The ACCESS DMD™ program will enable
participating physicians to obtain and provide deflazacort*, an investigational medication for eligible U.S.
patients diagnosed with DMD, while it is under development. ACCESS DMD™ participants will be
provided with deflazacort* at no cost while participating in the program and it will be sent directly to
patients or their caregivers.
Deflazacort* is an investigational glucocorticoid that has not been approved by the FDA for any indication
and is therefore not proven to be safe and effective. The sponsor, Marathon Pharmaceuticals, LLC, plans
to submit a New Drug Application to the FDA seeking approval of deflazacort* for the treatment of DMD.
Deflazacort* is currently under investigation as a potential treatment for DMD in both pill and liquid
formulations. Deflazacort* is approved for use in a wide range of indications in Europe and Latin America,
however, it is not approved for DMD in any country in the world. Based on published literature, its potency
is approximately 70 to 90% of prednisone; 6 mg of deflazacort* has approximately the same antiinflammatory potency as 5 mg of prednisolone or prednisone.
I am currently participating in the ACCESS DMD™ program and I invite you to consider referring patients
who may qualify for this program or consider registering yourself online as a participating physician.
Please read on for a summary of the eligibility criteria¥:

The patient must have a diagnosis of Duchenne muscular dystrophy determined by appropriate
signs and symptoms, including onset of weakness before 5 years of age and/or formal diagnostic
confirmation (e.g., muscle biopsy and dystrophin analysis, or DNA mutation analysis).

The patient is ≥ 5 years old.
Page 1 of 2
224880 USA Recruitment 20150827 Dear Physician Letter English 3.0
ACCESS DMD™: An open-label, expanded access protocol intended to provide treatment with
MP-104 (deflazacort)* to U.S. children, adolescents, and/or adults with Duchenne muscular
dystrophy

A steroid-naïve patient <8 years of age, if treated at a FOR-DMDa site, must have
documentation to support that he was presented with or was considered for the
FOR-DMD study, but was determined ineligible, unable, or otherwise unwilling to
enroll. The patient must be unwilling or unable to enroll in any other clinical studies
examining the efficacy of deflazacort.

Child or adolescent patient (<18 years) weighs at least 13 kg and has a body mass index (BMI)
≤40 kg/m2. Adult patient (≥18 years) has a BMI ≥18.5 and ≤40 kg/m2.

¥There
Child or adolescent patient is up to date on all childhood vaccinations, including varicella vaccine
(chicken pox).
are additional eligibility criteria to determine if a patient will qualify for program participation.
a
The FOR DMD study is an international, multi-center study intended to compare the benefits and side effects of the
three most widely prescribed steroid treatments in children with Duchenne muscular dystrophy (DMD). The FOR
DMD study intends to compare daily prednisone (0.75 mg/kg/day), intermittent prednisone (0.75 mg/kg/day, 10 days
on, 10 days off), and daily deflazacort (0.9 mg/kg/day). For additional information, visit www.clinicaltrials.gov,
NCT01603407.
There are no program required visits. Participating physicians will follow standard of care for the
management of DMD. Patients may continue participating in ACCESS DMD™ until one of the following
occurs: (a) deflazacort* is approved in the U.S. (if approved) and made commercially available; (b) the
ACCESS DMD™ program is terminated; (c) you determine that the patient should stop taking part in the
program; or (d) your patient (or their caregiver) decides that they (or their child) no longer wish wants to
participate.
Please contact me if you wish to discuss referring patients to ACCESS DMD™ in greater detail. If you are
interested in registering online as a participating physician, you can find additional information and the
tools to get started at www.accessdmd.com.
I look forward to speaking to you soon.
Yours sincerely,
[Participating physician NAME] for the ACCESS DMD™ program
[Include telephone numbers and e-mail address]
*Deflazacort is an investigational medication that has not been approved by the Food and Drug
Administration (FDA) and is therefore not proven to be safe and effective.
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224880 USA Recruitment 20150827 Dear Physician Letter English 3.0