PPMD was recently joined by the team at PTC Therapeutics to discuss upcoming changes and continuing community support around EMFLAZA (deflazacort). EMFLAZA came on the U.S. market in 2017 as the first FDA-approved steroid specifically indicated for the treatment of Duchenne. Since then, it has been available exclusively through PTC. However, earlier this month, patents protecting PTC’s exclusive rights to deflazacort expired. 

Generic deflazacort

With this loss of exclusivity, other drug companies can now distribute generic deflazacort in the U.S. As of February 2024, no companies currently offer generic deflazacort, but we expect this may change in the coming weeks and months. As a reminder, per FDA regulations, generic medications must demonstrate equivalency to their branded counterparts. You can learn more about brand vs. generic regulations and more here.

Dispense as written

If you or your child is currently on EMFLAZA and would like to stay on brand medication, you can request that your medical provider write a “DAW,” or “Dispense as Written” code on the prescription. This tells the pharmacy they must dispense brand EMFLAZA and may not dispense generic deflazacort. However, your insurance may have restrictions to their coverage of brand-name medication. If you want to stay on brand name EMFLAZA, you can contact PTC Cares to ensure your insurance information is up to date and work with them to utilize patient assistance and co-pay programs.

Helpful resources

With the steroid landscape in Duchenne changing rapidly, you have many options to choose from. If you are considering changing your steroid regimen, switching from brand to generic medication, or have other questions, we encourage you to speak with your neuromuscular provider. You can also check out PPMD’s recent webinar and FAQ here.

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