Like any of you who participated in today’s Advisory Committee Meeting at the FDA for PTC Therapeutic’s ataluren, I am emotionally exhausted. Whether you participated by speaking during the Open Public Hearing, attending the Ad Comm on the FDA campus, submitting your written testimony, or streaming the meeting from home – I would imagine that you, too, are drained from the hours of robust discussion.
We are starting to become pros at this, the Duchenne community. And if you were part of another rare disease community, you may be envious that we continue to get these opportunities in front of regulators to share our stories. But that doesn’t make it any easier. So much rides on each of these meetings, so many lives affected.
Today was important for the entire community. Whether or not you or your child has a nonsense mutation, we all want a victory for our families. Today, we did not learn of a final outcome. That decision likely won’t come until on or about PTC’s PDUFA date of October 24, 2017. Today was another critical layer in the product review process. Today was the opportunity for three dimensions of the product experience to be explored: the applicant’s analysis (PTC), the regulator’s review of the data (FDA), and the patient community’s experience with the product (our community’s lived experience on ataluren).
Today’s process was critical and was exactly what PPMD and our community have fought to be included in for decades. Today was the day that the data is provided in context. In the context of Duchenne. Even though we may feel worn down by today, we need to recharge our batteries and, like always, continue to fight for the approval of therapies we believe so that every single person in our community has a potential treatment for Duchenne.
Thank you to the families that participated in today’s meeting. Your testimony, as well as the written testimonies of those families who submitted prior to the Ad Comm, had a tremendous effect as indicated by almost every committee member.
Ataluren Ad Comm Discussion & Voting Results
Today’s final discussion and vote are similar to scenarios we discussed in a prior webinar. The question was included in briefing documents the FDA published Tuesday. Only one question was presented with a discussion followed by a vote on that discussion. The framing of the question allowed for the Advisory Committee to indicate whether the data presented were adequate enough to be conclusive of efficacy, or whether additional studies were needed to in order to be conclusive.
The question, as well as the votes, were designed to facilitate discussion around the findings and complexities of each of the studies that had been presented throughout the day, and to potentially map out a path forward.
It is important to remember that the result of these votes do not reflect the final decision of the FDA. Discussions from today’s Advisory Committee Meeting are used to help further inform regulators before a decision is made, on or prior to the PDUFA date of October 24, 2017.
Questions from Advisory Committee following Open Public Hearing (OPH) – prior to voting:
Question posed by FDA for the Advisory Committee:
1. VOTE: The best interpretation of the information presented today regarding the use of ataluren for the treatment of dystrophinopathies resulting from nonsense mutations in the dystrophin gene is that:
Question clarified that OPH testimony as to be included in the ‘totality of the data’ to be considered in the vote.
Summary of themes from discussion of the vote:
Open Public Hearing:
PPMD has connected with PTC to find out how we can help them between now and the PDUFA date of October 24, 2017. We will continue to work with the FDA to help better the review process in any way that makes sense. And regardless of today’s meeting or the final outcome, we will continue to work with families seeking access to ataluren. Stay tuned for ways you and your family can help.
In many ways, PPMD and PTC have grown up together. PTC has been invested in the Duchenne community for over a decade and today was an opportunity for that history to be shared with the FDA. Families that have been participating in this trial have children – teens and young adults now – who have been on ataluren most of their lives. Think about that. These kids have been on ataluren longer than they haven’t.
Thank you to the heroes of the day – the young men and parents who shared their intimate and long history with this drug. PPMD will continue to bring you updates about this therapy as it becomes available.