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:

  • Novel trial designs that recognize the challenges inherent in rare disease populations like Duchenne;
  • Conditional approval or similar routes to speed access to patients while additional studies are conducted; and
  • Helping agency leaders and reviewers better understand patient and parent perspective on the important question of benefit/risk thresholds.

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:

  • There are many important natural history studies underway, each of which helps inform clinical trial design and interpretation. Some people in our community have pointed to a specific set of data as “problematic.” These and other natural history data sets are valid, important data that result from the hard work and dedication of multi-center scientific teams. There are ongoing natural history data efforts funded by a 5-year National Institute of Health (NIH) research grant, the DOD, and the National Institute of Disability and Rehabilitation Research (NIDRR). The goal of natural history studies is to better understand Duchenne and clinical outcome measures, and not to help one specific company or another.

  • This data have been presented in multiple public sessions including those attended by both the European Medicines Agency (EMA) and the U.S. FDA. In June 2013 the most recent available natural history data for Duchenne were presented to the EMA, the academic community, and most pharmaceutical companies working in the Duchenne space (including Sarepta) were in attendance.

  • The natural history studies in progress are critical to eliminating the need for a placebo arm of clinical trials, a key component to helping accelerate the pace of clinical trials to ultimately help accelerate the pace of review.

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.

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Comment by Anup Kalra on November 14, 2013 at 11:26pm

Can we request FDA that no more placebo controlled trials as natural history data is available now, And no more biopsies as dystrophin is not the correct bio marker for functional improvements. At least save kids from any further torture (may be a harsh word but is truth of clinical trials) if we are not in a position to give any relief.

Comment by Andrea Cleary on November 14, 2013 at 12:19pm

I am tired of it.

This life, this day.

Fess up ? Shut up.

Fed up ? Throw up.

Projectile puke this weariness

From my core.

Limbs leaden, mind is mud

Yet sleep still exhaustingly illusive.

Eyes involuntarily fluttering

Like a butterfly in a fan.

Sounds never sensed, auditory tricks,

Startling resistance awake.

Pleading for waves of warmth

To lap over me.

Sweet dreams please carry me home,

Away from this shore, this view.

I am tired of it,

Can't stand (it) anymore.

Tonight's glass half empty will be

Tomorrow's half full.

So tired.

Comment by Neil McLaughlin on November 14, 2013 at 5:41am
but maybe it should be more positive in the long run.
Comment by Neil McLaughlin on November 14, 2013 at 5:37am
I know this is really disappointing but it also seems to me that with recent failures coupled with ptc in 2010, the end points need to be changed and what is the point of spending the next 2-3 years with phase 3 studys that are destined to fail because the end points are not correct. clearly these drugs work, or are clinically beneficial. the fda are bound by law and data not opinion, it is annoying, upsetting and demoralising but it must be done properly to succeed. they havent said go away it doesnt work, they have suggested end points need to be modernised to allow for the better care given these days. I thought the glass was half empty when I first read this
Comment by Andrea Cleary on November 13, 2013 at 9:54pm

Hear, hear!


Comment by Joanna Johnson on November 13, 2013 at 9:40pm

Thank you for speaking the truth.

Comment by Debbie Dupree on November 13, 2013 at 3:56pm

Amen!  Pat you are a hero for the DMD community!

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