PPMD Submits Community Sign-On Letter to FDA Supporting Approval of Ataluren

PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.

Earlier this month, PPMD reached out to FDA requesting guidance on the best mechanism for patient input during the formal dispute process with PTC. We have learned that there is no formal process to submit such information.

We understand that the formal dispute resolution is a regulatory process that concerns confidential information being exchanged between the agency and the sponsor and that there is no formal mechanism for patient engagement within the process of a dispute resolution. We respect the process underway and have requested that FDA continue to consider patient-experience data from Duchenne caregivers throughout this process. 

To that end, PPMD submitted a community sign-on letter reflecting the collective support of the approval of ataluren based on direct experience with ataluren. The letter of support was accompanied by data collected by PPMD through a patient experience survey in an effort to fully understand the experiences of individuals and their caregivers who had direct experience with ataluren. Sixty-one families participated in the survey, eager to convey real world evidence and first- hand observations related to benefit and risk. All respondents believed ataluren to be safe and most suggested they saw benefit in terms of slowing progression.

PPMD will continue to actively engage in the review process and will communicate all developments and opportunities for continued impact and engagement.


Ataluren Group Letter

Patient Experience Survey Results

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