The U.S. Food and Drug Administration (FDA) in August accepted Marathon Pharmaceuticals’ New Drug Application for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. A decision on deflazacort, which previously has received Fast Track status, Orphan Drug designation and Rare Pediatric Disease status from the FDA, is anticipated in February 2017.
FDA approval of the two NDAs, one for an immediate-release tablet form of the experimental treatment and one for an oral-suspension (syrup) formulation, would allow widespread access to deflazacort for kids and adults with DMD across the United States and make the drug among the first FDA-approved treatments for Duchenne.
An approval also would change the way people, who currently may be ordering deflazacort from outside the United States or getting it through Marathon’s expanded access program, are able to access the drug.
For those who currently order deflazacort through Masters Pharmaceutical
If you currently obtain deflazacort through Masters Pharmaceutical, you should already have received a letter from the company containing details about how long it will be able to continue supplying the drug to people in the U.S.
Should the FDA grant approval to deflazacort, Masters will be able to continue to supply you with deflazacort as it currently does (with a maximum 3 months’ worth of medication) until Marathon’s drug becomes commercially available in the U.S. At that time, Masters will end importation of deflazacort into the country.
Marathon has set up a Deflazacort Transition Program in advance of U.S. approval of the drug to help smooth the transition for those who currently are receiving deflazacort through Masters. To register, visit www.deflazacort.us.
For those who are receiving, or who wish to receive, treatment with deflazacort via EAP prior to U.S. approval
During the FDA review process, Marathon is making deflazacort available to people with Duchenne in the U.S., at no cost, through AccessDMD, an expanded access program (EAP) operating under FDA authorization.
If your physician is registered with AccessDMD, you may wish to consider enrolling in the EAP and begin receiving deflazacort for free. You must contact your physician to begin the process.
If your physician is not currently registered in Marathon’s EAP, the anticipated FDA decision on potential approval makes it unlikely that there is sufficient time for you to participate, but you can still register with Marathon at www.deflazacort.us to ensure timely access to deflazacort if and when approval of the drug is granted.
Following an FDA approval, the EAP will continue for an extended period of time to supply deflazacort to patients while they are transitioning to commercial supply. By opting into the transition program, you will be connected to Marathon’s Patient Support Services team as soon as possible to ensure the continuity of your drug supply.
Patients, families and physicians can learn more about AccessDMD, including a list of clinical sites participating in the program, by visiting www.AccessDMD.com or calling (844) 800-4363.
Obtaining deflazacort after FDA approval
If the FDA grants approval to deflazacort for the treatment of DMD, you will need to contact your physician in order to get a prescription for the drug. To ensure the process goes as smoothly and efficiently as possible, you can register now with Marathon’s Deflazacort Transition Program at www.deflazacort.us.
Marathon is planning to make deflazacort available as quickly as possible following an FDA approval. If the drug is not available immediately upon approval, Masters will continue its importation of the drug until Marathon’s commercial supply is available for sale. If you are a Masters customer or EAP participant, you may opt into the Deflazacort Transition Program, which will connect you with a patient services group immediately upon approval to ensure the continuity of your drug supply.
If deflazacort is approved, your physician will need to write a new prescription for you. Deflazacort will be made available from Marathon’s U.S. Patient Support Services team. It will not be available at your local pharmacy.
Deflazacort may delay disease progression
Deflazacort, a glucocorticoid, works as an anti-inflammatory and immunosuppressant. In the United States, the drug is considered an investigational therapy as it has not been approved by the FDA for any disorder. Versions of deflazacort are approved in other countries, but not for treating DMD.
In a pivotal study involving 196 participants with DMD, results showed that treatment with deflazacort improved muscle strength. Side effects reported to date for the drug include cushingoid appearance (puffiness and weight gain), abnormal hair growth, skin redness, swelling of the nasal passages or upper respiratory infection, irritability, and cataract formation.
MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including deflazacort, in DMD, with studies to determine the drug’s mechanism of action, effects, side effects and best dosing regimen. Through its basic research program, MDA currently is supporting development of biomarkers for glucocorticoids such as deflazacort with a grant to Yetrib Hathout for $300,000, and research by Eric Hoffman to understand the effects of glucocorticoids on muscle repair with a grant for $321,659.