PTC Therapeutics Announces Agreement to Acquire Emflaza™ (deflazacort)

Today we learned that PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals, LLC to acquire all rights to Emflaza™ (deflazacort). Emflaza is the first treatment approved in the United States for all Duchenne patients five years and older, regardless of their genetic mutation.

PTC has a long history with our community. And it is this long history and deep understanding of Duchenne that gives us hope that this transaction will truly expand access of deflazacort in the U.S. For decades, PPMD has wanted options for families including deflazacort as a steroid option.

Today, a new conversation begins with a partner we know well. PPMD looks forward to this new chapter in our relationship with PTC. But even though the players may have changed, our concerns and questions remain the same and our recommendation for a path forward remains unchanged.

Shortly after announcing a pause in the commercialization of Emflaza by Marathon in mid-February, PPMD recommended that Marathon seek input from a broad array of stakeholders including Duchenne clinicians, health insurance plans, pharmacy benefit managers, and Duchenne advocacy groups.

Immediately after this morning’s announcement, we spoke with PTC. We relayed that we believe that this is an important inflection point for our community. PTC now has a unique opportunity to elicit evidence-based inputs (a combination of collected data and real world experience) from stakeholders that could be used to help inform their pricing, as well as ensure that they are creating a favorable access environment for the Duchenne community’s access to deflazacort and other therapies. We further urged PTC to be transparent about the methodology they will use when establishing their pricing.

Concerns we are urging PTC to consider include:

  • Will the price of Emflaza cause families to see an increase in their health insurance premiums?
  • Will the price of Emflaza lead health insurance providers to first recommend the less expensive alternative, prednisone?
  • We believe that treating Duchenne will take a combination of therapies. Will the revised price of Emflaza allow for a favorable access environment to the combinatorial therapy approach in Duchenne?
  • Many physicians recommend steroid intervention to begin in children under 5 years of age. The Emflaza label is for children 5 years and older. How will PTC provide an access pathway to Emflaza for patients under age 5?
  • What happens to EMFLAZACares? What patient assistance will be available?
  • What if you have Becker muscular dystrophy and require steroid medication? Is there an access pathway to Emflaza?
  • What does this acquisition mean for families overseas? Either those on Translarna or those on deflazacort?

The approval of a drug in Duchenne is an incredible moment for our community. We are cautiously optimistic that today’s news signifies that PTC will ensure that deflazacort will be made available as a therapeutic option to our entire Duchenne population, including families that were unable to import it from overseas. We are grateful to PTC for their continued commitment to our Duchenne community, as well as to ALL of the 40+ industry partners that are joining us on the battlefield and developing therapies that could one day end Duchenne.

PPMD is committed to ensuring that the therapies approved in our community are truly accessible and truly affordable, without jeopardizing the decades of landscape-changing work we have done together. We look forward to engaging with PTC as they move Emflaza forward.

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