Frequently Asked Questions

  1. 1 What is the purpose of an Expanded Access Program (EAP)?

    The intent of an EAP 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 FDA has authorized the use of deflazacort* for eligible patients with DMD under the ACCESS DMDTM program. For more information to share with your qualifying patients about EAPs, please visit Expanded Access Program Information for Patients.

  2. 2 What is the difference between an EAP and a clinical study?

    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 DMDTM, it still carries some of the same regulatory requirements of a clinical trial.

  3. 3 Is everyone diagnosed with DMD eligible for this EAP?

    No, please refer to the full criteria. All patients and/or their caregivers interested in taking part in the program are being encouraged to speak to their physician about the possibility of enrolling.


  4. 4 What is deflazacort*, the investigational medication that is being offered?

    An investigational medication is a medication that is still in research and development and that has not been approved by the FDA for the proposed use under study. Deflazacort* is an investigational glucocorticoid that has not been approved by the FDA 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 a glucocorticoid that is not approved for any indication in the U.S., but 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.

  5. 5 How is deflazacort* administered?

    Patients enrolled in ACCESS DMDTM will receive oral tablets (6, 18, 30, 36 mg strengths) or oral suspension (22.75 mg/mL strength) in an open-label fashion. The participating physician will determine whether the patient will receive oral tablets or oral suspension. A specialty pharmacy will deliver an adequate supply of deflazacort* based on the participating physician’s order of dose and regimen directly to the patient or caregiver.

  6. 6 Where can I learn more about deflazacort*?

    You can access the current deflazacort* Investigator’s Brochure through the online portal once you have registered for the program and signed the confidentiality agreement. If you have further questions about deflazacort* please call: +1-844-800-4DMD (4363) or email mailto:

  7. 7 Will I still be responsible for my patient’s ongoing care if they take part in the program?

    Yes, once participating you will still retain the same responsibilities for your patient’s ongoing and usual standard of care. You will also be subject to additional regulatory responsibilities as an investigator (see FAQ, “What are the regulatory requirements for an EAP?”).

  8. 8 Can my patient participate in other clinical studies while being in this program?

    Patients participating in ACCESS DMDTM cannot participate in any other clinical studies involving deflazacort*. However, patients who are participating in clinical studies for other investigational DMD treatments/therapies may still be eligible for participation in ACCESS DMDTM.

  9. 9 If my patient is eligible, but I do not want to participate in the program, is there a way for my patient to still take part in the program?

    Yes. You and your patient can review the list of participating physicians in your area and decide if it is a good option for you to transfer your patient’s care for this program to an alternate physician who is currently participating. Check the list of participating physicians to see who is currently participating.

  10. 10 How long will my patient take part in the program?

    Patients may continue participating in ACCESS DMDTM until one of the following occurs: (a) deflazacort*, is approved in the U.S. (if approved) and made commercially available; (b) the ACCESS DMDTM 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 wants to participate.

  11. 11 What are the expectations for data collection in the program?

    This program will only collect safety and tolerability data during the program in accordance with the ACCESS DMDTM protocol and applicable regulatory requirements.

  12. 12 What is the program visit schedule?

    There are no program required visits. Participating physicians will follow standard of care for the management of DMD.

  13. 13 What are the regulatory requirements for an EAP?

    As a participating physician under an EAP, you are subject to the same regulatory responsibilities as other investigators in clinical studies, and as outlined on the Form FDA 1572. For example, you will be required to obtain informed consent from your participants and are subject to adverse event reporting requirements and record keeping obligations. Any specific responsibilities will be outlined in the EAP protocol. Institutional Review Board (IRB) oversight is also required for EAPs. You may either submit your regulatory package to an IRB of your choosing or to the identified central IRB.

  14. 14 What is the role of the specialty pharmacy in this program?

    A specialty pharmacy is different from traditional pharmacies in that it helps coordinate many aspects of patient care and disease management. For the purposes of this program, the specialty pharmacy will be involved in deflazacort* delivery and return.

  15. 15 Who will have access to my patient’s information collected during participation?

    Any personally identifiable information (for example, contact details required for shipping and distributing the investigational medication) will not be accessible to anyone who is not directly associated with ACCESS DMDTM, except with the patient or caregiver’s permission or as required by law.

    Any information that is gained from this EAP may be used for publishing outcomes by the sponsor. However, this information will be combined with other participant data and would not be used to identify any given participant.

  16. 16 What happens if deflazacort* is approved and becomes commercially available?

    If deflazacort* obtains FDA approval, approximately 90 days prior to commercial launch of deflazacort*, the sponsor or the sponsor’s representatives will begin working with you and your patient(s) in an attempt to ensure a seamless transition of your patient(s) to the approved product.

  17. 17 How can I obtain a copy of the EAP protocol and consent forms for review?

    After you have registered for ACCESS DMDTM and signed a confidentiality agreement, you will have access to the EAP protocol, Informed Consent documents, as well as other program specific documentation for review.

  18. 18 Who can I speak to if I still have questions?

    If you have further questions about this program please call: +1-844-800-4DMD (4363) or email

*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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