About ACCESS DMD™

ACCESS DMD™ is an expanded access program for children, adolescents, and adults in the U.S. diagnosed with DMD. We invite you to enroll your eligible patients with DMD in the program.

  • 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.
  • Expanded access is a general term often used to refer to a variety of regulatory mechanisms available to provide investigational treatment access to patients with life-threatening or severely debilitating disease for whom commercially available therapies have failed or are not available. By definition, compassionate or expanded access programs (EAPs) are intended to address unmet need and, as such, beyond the regulatory requirements for a given country, no two expanded access programs are alike. The distinction between an expanded access program and a clinical trial is that the use of an investigational medication in an expanded access program is intended for treatment, not to answer safety and effectiveness questions about the investigational medication. The use of an investigational medication in a clinical trial is intended for research (systematic collection of data with the intent to analyze it to learn about the medication). While the collection of data is minimal for ACCESS DMD™, it still carries some of the same regulatory requirements of a clinical trial.
  • 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.
  • The FDA has authorized the use of deflazacort* for eligible patients with DMD under the ACCESS DMD™ expanded access program.
  • As with any investigational medication that has not been approved by the FDA, deflazacort* may or may not be effective in treating your patients’ DMD and there may be risks associated with its use.
  • ACCESS DMD™ participants will receive deflazacort* at no cost while participating in the program.
  • Once ordered by an ACCESS DMD™ participating physician, deflazacort* will be sent directly to enrolled patients or their caregivers. Costs for other tests or routine medical care will need to be covered by the patient’s insurance or at the patient’s expense and are not reimbursed by the ACCESS DMD™
  • Not all patients with DMD who wish to participate in ACCESS DMD™ will qualify. Several criteria detailed in the ACCESS DMD™ protocol must be met in order for patients to participate.

For more information about expanded access programs to share with your patients, please visit Expanded Access Program Information for Patients.

*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.

Physicians are invited to register patients who fit the summary of eligibility criteria¥ below:

  • 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.
  • 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 also 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.
  • Child or adolescent patient is up to date on all childhood vaccinations, including varicella vaccine (chicken pox).

¥There 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.

About Deflazacort*

Deflazacort* is an investigational glucocorticoid that has not been approved by the FDA for any indication and is therefore not proved to be safe and effective. 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 anti-inflammatory potency as 5 mg of prednisolone or prednisone. More information about deflazacort* will be made available after you register as a participating physician in ACCESS DMD™.

How do I participate?

If you are interested in participating as a physician for the ACCESS DMD™ program, follow these simple steps:

1) Check the criteria to see if you have potential patients that would qualify to participate in the program.

2) Click here to connect to the registration website which will allow you to register yourself and any eligible patients who agree to enroll in the program.

*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.

Locate Participating Colleagues!

LIST OF PARTICIPATING PHYSICIANS