Letter to Duchenne Community from PTC Therapeutics

We are happy to share the following letter to the Duchenne Community from Stu Peltz, CEO PTC Therapeutics. 

March 24, 2017


Dear Duchenne Community,

We were very excited to announce on March 16, 2017 that PTC Therapeutics entered into an agreement to acquire the FDA approved steroid for Duchenne muscular dystrophy, Emflaza™ (deflazacort) from Marathon Pharmaceuticals. We anticipate closing the transaction in the second quarter. This acquisition continues our nearly 20 year partnership with the Duchenne community to address the unmet medical need of this devastating disorder and to bring treatments to fundamentally change the lives of those living with Duchenne.

Based on our long-standing global experience with DMD, and our strong partnership with the community, we believe PTC is uniquely positioned to launch Emflaza in the US. We will build upon this expertise and our experienced US commercial leadership to enable a successful launch to bring Emflaza to patients. We are excited to introduce Emflaza to the U.S. market following the closing of the planned acquisition. We understand there are a number of questions regarding the launch of Emflaza. While many elements of our launch are still being finalized, we believe it is important to share the information that we have available at this point in time.

We understand that availability and access are important for the community. It is critical that families can afford medicines without negatively impacting their family’s financial health. As we develop pricing and launch plans for Emflaza, we will incorporate this and additional feedback from the Duchenne community, patient advocacy organizations and other related stakeholders into the plan. We are committed to making product availability a priority.

Between now and the closing of the transaction, we are focusing on engaging with the community as your insights are very important to us. The input that the Duchenne community provides will help to shape our commercial launch plans. We plan to share the specifics of our plans following the closing. Please continue to communicate your insights with patient advocacy group leaders.

It is our expectation that patients will continue to receive Emflaza through the Expanded Access Program and order from Masters Pharmacy until the close of the transaction. Healthcare Professionals may continue to write for Emflaza and submit prescriptions to EMFLAZACares for processing. We are committed to providing a smooth transition at the time of launch.

Thank you in advance for your support during this transition. By addressing inflammation, Emflaza is key to preserving muscle function and ultimately slowing disease progression. In keeping with the mission of PTC, we are excited to work with the community to bring this therapy to Duchenne patients.


Stuart Peltz

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