A note from Debra

Deflazacort Transition Program Reminder

Posted on January 17, 2017 6:42 pm


Marathon submitted New Drug Applications (NDAs) to the Food and Drug Administration (FDA) last June for deflazacort. The NDAs have been accepted for filing and review. The FDA expects to complete its review of the applications in early February. If approved, deflazacort will become accessible in the U.S. and it would be the second treatment approved for Duchenne in the United States and the first indicated for all Duchenne patients.

We hosted a webinar in November that highlighted the regulatory process to date, Marathon Pharmaceuticals’ Expanded Access Program (EAP) for deflazacort and what a potential FDA approval of deflazacort would mean in terms of access to the drug in the United States. It included steps current deflazacort patients might choose to take to help ensure access to U.S. supply of deflazacort if FDA approval is granted.

You can learn more about the Deflazacort Transition Program at  https://deflazacort.us/.

  • Deflazacort Transition Program is designed to provide support for patients already taking deflazacort supplied either through Marathon’s EAP or by Masters
  • Continuity of supply for EAP and Masters patients during the transition can be facilitated by voluntarily opting in to Marathon’s Deflazacort Transition Program
  • Patients obtaining deflazacort from other sources may inquire more generally about upcoming changes via the Deflazacort Transition Program

If you choose to sign up for the Deflazacort Transition Program, you will be notified if and when deflazacort is approved in the U.S.

We need your help to advance a treatment to clinical trials for rare, Duplication 2 mutations!

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