Yesterday was a disappointing day for all parents, grandparents, aunts, uncles, and others touched by Duchenne. Like most of our community, I awoke to the news that multiple factors have led Sarepta to announce that it would delay the filing of its much-anticipated new drug application for eteplirsen with the Food and Drug Administration. Coming on the heels of a late-stage failure in the drisapersen trial, it is understandable that this news would make our community frustrated and even angry.
As parents and loved ones impacted by Duchenne, we know what bad news is. We also know the importance of meeting adversity head-on. The time is now for our entire community to come together, to understand thoroughly the many challenges that led to this decision, and to commit to confront them head on.
Nearly 20 years ago, I joined other parents impacted by Duchenne to form Parent Project Muscular Dystrophy with the single goal of ending Duchenne. I understand that discouraging news leads people to take certain action and say certain things, and it has come to my attention that a small minority in our community has chosen to lay the blame for this most recent setback on PPMD. Such accusations are baseless, hurtful, and counterproductive. When false accusations have arisen in the past, I have chosen to dismiss them so we can continue focusing laser-like on the goals of ending Duchenne and improving care and quality of life for patients. However, this current attack cannot go unanswered.
Advancing safe and effective treatments and therapies is impossible to accomplish in the United States without the engagement of the FDA. FDA review of potential therapies is a highly complex set of issues. For several years, PPMD has been engaging with Congress and with senior leaders at the FDA to help provide the agency with the tools, information, and resources they need to review potential candidate Duchenne therapies as quickly as possible. Our engagement with FDA has focused on many issues including:
The latter involved the first-ever scientific study of our community on benefit/risk perspectives, a project that involved many of you. This data was seen by FDA as being exactly the type of information the agency needs to understand in discharging its work.
A few critics are laying blame at the FDA’s analysis of natural history data, information pointed to by Sarepta as influencing its decision to delay filing. Here are some important facts that may have been overlooked:
Many in the community are looking where to point the fingers and we see in some cases the community shooting inward instead of rallying to support one another. Rather than spreading untruths and finding scapegoats, our community should be uniting to better understand the points raised by FDA and to respond as strongly and as swiftly as possible. Our community – particularly our sons – deserves better than petty infighting, and PPMD is ready to work with all partners to move this ball forward. This is not our first setback, and it mostly likely will not be our last. But if we fail to focus on the end goals, we will not succeed.
Thanks to the efforts of so many, the future today looks more promising than ever, but as this news underscores, much more work remains to be done. PPMD’s interest is not in one potential therapy over another or one entity over another. Our interest is in ALL therapies to treat Duchenne, and we will continue to support development efforts across the sector and to lead community engagement with FDA.
Next week, we will host a webinar with Craig McDonald, MD to discuss the available natural history studies he has been a part of and the data that has been collected by he and other investigators. Click here for more information about this webinar.
In just a few weeks, we will be convening a public policy summit with FDA, a public event to be held in the Washington area. I encourage as many members of the community as possible to attend this meeting, to make your voices heard, and to work with PPMD and others to advance constructive solutions to these challenges. By working as one community united, we can accomplish so much more than we could if divided.
I am confident that this community – each and every one of you – knows well what I stand for. I was driven to this mission in life not by choice. Like most of you, I was driven by the desire to save my sons. While that goal is no longer possible, I commit myself each and every day to our shared goal of ending Duchenne.