PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access

Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.

On today’s call, we learned:

  • Pricing for EMFLAZA will start at $35,000 a year for a child that weighs 25kg (approximately 55lbs)
  • A weight-based pricing model is more relevant to the Duchenne community PTC believes
  • EMFLAZA will launch in the U.S. “in the coming weeks”
  • Progress was already being made with payers, prior to the transaction between PTC and Marathon
  • PTC will also share their own data to payers
  • PTC’s goal is that all eligible patients have access to EMFLAZA, irrespective of insurance or ability to pay
  • They will provide customized, personalized support for all patients via EMFLAZACares
  • Start form and details about receiving EMFLAZA found at EMFLAZA.com or by calling 844-EMFLAZA
  • You must submit start form to benefit from the EMFLAZACares program
  • There is immediate access to a bridge program if you are receiving EMFLAZA from the Expanded Access Program (EAP) or Masters
  • You will receive from the EAP and Masters until commercially available
  • PTC believes patients on EXONDYS 51 will be covered for both because they are complementary therapies
  • PTC’s goal is $0 for patients or as close as possible
  • Patients under 5 and Becker patients: Physicians can submit the start form and payers will decide whether or not to pay, with specialty pharma as adjudicator

We know that, like PPMD, many of you are very concerned about the new price that has been announced. While the price is lower than the proposed Marathon price point there remains confusion about the weight based dosing and how that is tied to pricing. PTC also indicated that it anticipated that the process from point of start form initiation to payer access could take many weeks and likely require prior authorization.

This makes navigating access issues more important than ever. And as we are already doing with families who are navigating the complicated landscape of EXONDYS 51 access, PPMD will do everything we can to help assist families who want access to EMFLAZA. We will also work closely with PTC to better understand the progress made with payers to ensure a favorable access environment.

PPMD will update our online Access Resource to reflect EMFLAZA access, as information becomes available. For now, check out some of the useful general information we have pulled together.

Many Questions Remain

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

We hope that EMFLAZA will be that option, but we still have a number of outstanding concerns that we will address with PTC, including:

  • We need clarification about weight based dosing of Emflaza, and a potential maximum dosing per day per patient. There remains confusion around how this factors into the price.
  • 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?
  • What does this acquisition mean for families overseas? Either those on Translarna or those on deflazacort?

Stakeholder Engagement

As PTC mentioned on their public calls today, the company met with various stakeholders between the time the deal with Marathon was announced and today’s update. PPMD was one of the stakeholder groups that PTC met with. We shared with the company the importance of landing on a fair price for this therapy that was not only sustainable for the Duchenne community, but that protected future pricing in the rare disease space as a whole.

We reminded the company that pricing concerns may force families to make impossible decisions, choosing one therapy over another in a disease that we know will take a combination of therapies to eventually slow disease progression.

And we emphasized that once this drug goes to market, issues of access – in a payer landscape that has perhaps never been more uncertain – is a crucial concern. Families are changing insurance, losing benefits, and/or navigating new premiums multiple times a year in our community. We need to ensure that the people who need deflazacort the most are able to get it in a timely and affordable manner.

The price announced today does not reflect our conversations with PTC, nor the concerns we expressed. We hope that going forward, programs like EMFLAZACares will help families affordably access EMFLAZA without limiting coverage of other critical therapies currently approved as well as those in the pipeline.

PPMD will continue to monitor and remain engaged in the access environment in order to understand the impact of every new approval, providing materials and resources to help guide families.

Accessing Deflazacort from Other Sources

There a lot of questions about what this means for those who receive deflazacort from sources outside of the U.S. PPMD will be exploring available options and will share with the community our findings in the coming weeks.

For More Information

Visit EMFLAZA.com for more information about this therapy, as well as PTC’s patient assistance program, EMFLAZACares. For questions for PTC, please email the company at patientinfo@ptcbio.com.

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