The Future of Ataluren: A Hard Day But Still a Path Forward


Today the FDA issued a Complete Response Letter regarding PTC Therapeutics’ application for ataluren. Today’s news is hard. This decision is disappointing and not the outcome that our community hoped it would be. For more than a decade, our community has supported the development of this therapy. Hundreds of boys – some now young men – have dedicated over half of their lives to this trial. The community worked hard and sacrificed much.


However, today’s decision is not the end of the ataluren story. Today’s communication maps out a few paths forward and with those paths come continued hope for an eventual approval of ataluren in the U.S.


PTC reported that the FDA said, “an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness.” This path forward would not have been possible without the powerful involvement of our community throughout the regulatory review of ataluren. Because while we’re disappointed that there was not an approval today, we realized that this was always going to be a steep uphill climb. We knew the facts that the FDA had refused to file the application for the review of ataluren based on their assessment of the data package and that PTC had sought a full and fair review anyway. We knew that rarely has a company that has ‘filed over protest’ gone on to receive a full approval but that if any community could turn a ‘no’ into a ‘yes’, it was ours. So, we engaged. And that impactful engagement we did succeed in illuminating elements of ataluren’s potential impact that were not reflected in the original data.


PTC also indicated that they plan to file a formal dispute resolution request next week. This is unchartered territory for our community and PPMD will consult with both the PTC and our regulatory advisors about what we can expect from this process in terms of timeline and community involvement. As always, PPMD will share any new information we learn with you as soon as possible.


There is still a path forward for ataluren and for the hundreds of families that have participated in trials for more than a decade, as well as for the families waiting to obtain access to this therapy. This path is due in large part to the families who shared their direct experiences with ataluren through both oral and written testimony with the Advisory Committee. The powerful, patient-experience data brought forward by our community enabled the Advisory Committee and the FDA to recognize that there may be efficacy outside of what was measured within the clinical trials that is yet to be fully captured. Because you told your story, regulators have a better understanding of the potential of this therapy and are seeking trial data that reflects the experiences you shared. The Duchenne community’s voice was heard.



What’s next?


PPMD will work with PTC, doing what we can to support them while they develop a plan to collect the data the FDA has requested. We will learn more about the formal dispute resolution process and determine what role this community can play in that process.


It is critical that PTC continue to provide access to ataluren during this time and be cognizant of the subpopulations within our community who seek access. PPMD will work with PTC as they continue to support families benefiting from ataluren and those looking to participate. And while another trial was not the news we all wanted to hear, we are not starting at ground zero. PTC knows our community well, has over a decade of experience, and is currently collecting data from the global Duchenne community through both commercial access and a European clinical trial. We are grateful to PTC for their unwavering dedication to our Duchenne community.


So while today’s decision is a no to commercial approval for now, it is simultaneously encouragement for ongoing investigation and exploration and the idea that there is a path forward.


View the full press release from PTC Therapeutics, and stay tuned to PPMD for more information on how we can help optimize the regulatory pathway for all potential Duchenne therapies. 

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