In an effort to answer some of the questions that have arisen with the potential FDA approval of deflazacort in the U.S., PPMD has worked with both Masters and Marathon to put together the resource below, which we hope will provide clarity.
PPMD also recently hosted a webinar with Masters to learn more about what the transition process will entail. The recording is below.
What is happening with deflazacort?
In August, the Food and Drug Administration (FDA) accepted a New Drug Application (NDA) from Marathon Pharmaceuticals for the review of Marathon’s deflazacort. Marathon was granted a priority review and anticipates a decision by February 2017.
If approved, Marathon will market both an immediate release tablet and an oral suspension (liquid syrup) form of deflazacort
How will this effect deflazacort?
If the FDA approves Marathon’s deflazacort, it will be commercially available in the United States.
What should I do if I am now receiving deflazacort from Masters Pharmaceuticals?
If Marathon’s deflazacort is FDA approved in the US, it will no longer be possible (or legal) to import deflazacort from Masters. Until Marathon is fully capable of supplying drug to all patients, it is anticipated that Masters will be able to offer patients a 3 month supply of deflazacort as a “bridge” to the Marathon supply. Masters will work with patients to ensure that patients do not have an interruption in drug supply as they transition to Marathon’s deflazacort.
Should I do something now?
Yes. In anticipation of approval, Marathon has developed a Transition Program to assist with a smooth transition from Master’s to Marathon’s deflazacort. Patients now receiving deflazacort from Masters should register in the Transition Program at Deflazacort.us to help ensure a smooth transition between Master’s and Marathon’s deflazacort.
What will the Transition Program do?
A dedicated case manager from the Transition Program will contact you if the FDA approves deflazacort in the United States to discuss how you are obtaining deflazacort now and what your next steps should be.
What should I do if we would like to receive deflazacort through Marathon’s Expanded Access Program (EAP)?
Right now, through Marathon’s EAP, patients are able to access deflazacort free of charge. This EAP will be discontinued if Marathon’s deflazacort is approved.
If you would like to enroll in Marathon’s EAP, you will need to see a medical provider (physician, nurse practitioner) who is enrolled in the EAP program in order to gain access to deflazacort through the EAP. A full list of medical providers who are enrolled in the Marathon’s EAP can be found at AccessDMD.com (or by calling 844-800-4363).
A physician who is enrolled in the EAP will determine if you are eligible for the EAP and submit the paperwork required to enroll you. They will then submit an order for deflazacort to the specialty pharmacy, which will contact you and arrange for home delivery. If you are interested in participating in the EAP or have any questions about this process, please contact your medical provider, then visit AccessDMD.com or call 844-800-4363.
Patients who are enrolled in Marathon’s EAP may also want to consider registering with the Transition Program at Deflazacort.us. As above, a case manager from the transition program will contact you to discuss how you are obtaining deflazacort now. They will collect information and will alert you if deflazacort is approved and what your next steps should be.
What if I am not taking deflazacort now but I would like to start Marathon’s deflazacort if it is approved in the US?
If the FDA in the US approves Marathon’s deflazacort, you will need to get a prescription from your medical provider for this medication. Marathon is planning to make deflazacort commercially available as quickly as possible through their US Patient Support Services team. Marathon’s deflazacort will only be available to patients through central specialty pharmacies, and shipped directly to patients’ homes. You will NOT be able to get Marathon’s deflazacort through your local pharmacy. If you are taking prednisone now, a comparable dose of deflazacort can be prescribed.
Should I do something now?
Yes. In anticipation of approval of deflazacort, Marathon has developed a Transition Program. Patients who are not taking deflazacort now, but who would like to take deflazacort if it is FDA approved, should register with Marathon’s Transition Program at Deflazacort.us. After you register, a case manager from the Transition Program will contact you if deflazacort is approved by the FDA to discuss options that are available for the treatment of Duchenne, including deflazacort. They will collect information and will alert you if deflazacort is approved and what your next steps should be. Registration in this program will help you get access to deflazacort after approval, as quickly as possible.
How do I know that Marathon’s deflazacort is the same as Masters’ deflazacort?
Marathon conducted a bioequivalency study between its version of deflazacort and other versions of deflazacort currently available internationally. Those data, which showed that the versions were equivalent, were presented at PPMD's 2016 Connect Conference in Orlando and is available here.
Are there plans for patient support to help patients gain access to deflazacort?
Yes, Marathon does have plans to have a very comprehensive and robust patient support program. If you have questions, please call Marathon’s Medical and Scientific Affairs Department at 847-715-0699.