PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20

John Porter and I have been invited to participate in the FDA-NIH dystrophin methodology meeting on Friday, March 20. While we said yes, we realize this is a difficult and painful topic for all of us. Dystrophin feels like the brass ring, the best thing possible, the important foundation on which to build combination therapeutics.


From the moment we started the planning phase to write Draft Guidance, we recognized dystrophin was a challenging issue. In fact we tackled some of these issues in our recent dystrophin webinar. For parents and people with Duchenne, there is no argument and no challenge. The diagnosis of Duchenne is based on the absence of dystrophin. As parents we watch day after day what that means to our sons. As parents we have seen that florescent glow seen on muscle tissue sections, demonstrating the presence of dystrophin. And we are thrilled…it has taken a long time and this feels like a dream come true.

The dilemma is that companies focused on ‘foundational therapies’, those targeted to restoration of dystrophin are using different strategies to seek approval. We have seen the 6-minute-walk test data. We have seen stabilization which exactly coincides with our benefit/risk analysis: slowing progression is the highest priority. We know biopsies are difficult, painful and that many children have given tissues samples without knowing what was learned.


Within the guidance, we separated dystrophin as a separate working group – Biomarkers 1. We knew there were differences of opinion about how to best to quantify. Other discussions focused on what muscle to examine and what that might mean to people with Duchenne, acknowledging dystrophin as a biodynamic marker and the urgent need to qualify it as a surrogate.


FDA and NIH agree this is a difficult area and for this reason, decided to hold an open, public workshop. The discussion on Friday is limited to methodologies and how we, as a community, can come together, have a robust discussion, and agree. The purpose is to move the field forward.


According to the FDA and NIH staff convening this Friday’s meeting, the discussion is not about any specific drug. It is not about interpreting what that florescent glow on muscle tissue sections means in terms of functional improvement. We all believe having dystrophin expressed is likely to confer benefit. I have been told that that is not the question they want the panel to focus on this Friday.

Here is the goal. With so many different methodologies on the table, this meeting is focused on understanding methods currently available and driving toward consensus, one or more methods that we all agree will give us the information we need. It is about accelerating development and approval. It is about progress.


As a community we have lived with discussion, challenges, differences of opinion on methods of quantification. Together, we can change the landscape, by educating regulators, but also listening and learning. I am grateful that the FDA and NIH is willing to have this discussion and have opened their doors to include our community leaders.

This is about the future, our future, and our children’s futures.

Public Workshop 

If you would like to attend Friday’s public scientific workshop, click here for details. If you cannot attend but would like to watch a live stream of the meeting, click here Friday morning to attend virtually.

Note: The possibility of inclement weather has been forecast for Washington DC and surrounding suburbs beginning early morning on March 20. If the government announces a delayed opening (2 hours for example), the meeting time will also be delayed that amount of time. If the government opens on time, the meeting will begin on time. We will do our best to keep you updated and fingers crossed we don’t start Spring in the northeast with snow!

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