FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy

Today, my sons are dancing! Today we ALL celebrate the first FDA approval in Duchenne. Today the FDA approved Exondys 51 (aka eteplirsen) injection, specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with Duchenne.

Tears are all too familiar to the Duchenne community. But today, I am crying tears of joy. I landed in Boston, the home of the extraordinary Sarepta team and so many wonderful industry partners, and turned on my phone to this incredible news! It has been 32 years since my sons were diagnosed and after long last, it feels like my prayers are beginning to be answered.


Today is a historic day for the entire Duchenne community. It is a first step. But a huge step forward. Exondys 51 could potentially help 13% of the Duchenne population. We know that it will take a combination of therapies to fully halt the progression of the disease. There are still many people with Duchenne who won’t benefit directly from Exondys 51. But, what everyone in this community will benefit from is today’s first Duchenne approval from the FDA. Today, progress has been made that we believe opens the door for the dozens of therapies moving through the clinical trial process. Therapies that will help other mutations, other exons. Therapies that will experiment with new technologies. Therapies that will benefit all people with Duchenne.


For the last several months (years even), it seemed as if we would never get a win as a community. We thought we were doing everything right. PPMD convened the FDA with top thought leaders in the Duchenne space in December 2013. We pulled together stakeholders from across the Duchenne community to draft a guidance. We submitted benefit/risk data to the agency demonstrating that the highest priority to our community was slowing disease progression. PPMD and an extraordinary coalition of advocacy partners – The Race to Yes, the Duchenne Alliance, Jett Foundation, and the Center for Duchenne Muscular Dystrophy – orchestrated the largest community participation in an Advisory Committee Meeting ever. And still we waited. And waited. For well over 100 days.


With today’s accelerated approval comes questions, many of which we are already exploring and working to get you answers. In fact, for the last couple of weeks PPMD has been working with the team at Sarepta to schedule a webinar in the event of an approval that would dive deeper into what an accelerated approval means, labeling of Exondys 51, and what steps are being taken towards patient access and reimbursement. We hope to share the time and date of this webinar with you later this week. 


What we do know is that under the accelerated approval provisions, the FDA is requiring Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit. The required study is designed to assess whether Exondys 51 improves motor function of people with Duchenne with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. We also know that Sarepta received a rare pediatric disease priority review voucher, which comes from a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. This is the seventh rare pediatric disease priority review voucher issued by the FDA since the program began. You may recall, PPMD explained how significant these vouchers can be to companies that receive them and how we helped to make this happen!


None of today’s excitement would be possible without the sacrifices, bravery, and selflessness of all the young men and their families who participated, not just in the Exondys 51 trial, but in all of the trials that seek to end Duchenne or treat Duchenne. So many young men around the world willingly sacrifice their lives for the sake of progress and they must be thanked. They and their families are our true heroes.


We must thank the tireless efforts of the Sarepta Therapeutics team, under the leadership of Edward Kaye. They have been passionate and committed partners and have become part of our Duchenne family, getting to know our sons and caring for them like their own.


We celebrate the Congressional champions who have led legislative and policy efforts for us for decades that built infrastructure and paved the pathway that made today’s approval possible.


We celebrate the collaborative efforts of all of the Duchenne advocacy organizations who participated in this and other Advisory Committee Meetings, Congressional briefings, and meetings on Capitol Hill and with the agency to tell their stories. Today’s victory proves the power of our community coming together to serve a common purpose on behalf of our children. No one group or individual could have accomplished what we have accomplished…together.


And we thank YOU. We thank you for attending the Ad Comms for drisapersen and eteplirsen (both the original date and the snow date!). We thank you for sending in your written testimony, recording your video testimony, or participating in a group testimony. We thank you for sharing your story, however you chose to share it, so that the FDA could make this decision fully understanding the experiences of our community with these therapies.


You spoke. They listened.


There is still so much work to do. There is always work to do as long as families still receive a Duchenne diagnosis. Until all people living with Duchenne have treatments, PPMD will have work to do. But I think we’ve earned a moment to celebrate a victory decades in the making that EVERY SINGLE ONE in this community played a part in. And maybe I’ll drop by the Sarepta offices with a bottle of champagne to toast Ed Kaye and team. And thank them for their perseverance in the fight to end Duchenne.


Yes, today my sons are dancing indeed.

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Comment by Rahul Deshpande on September 21, 2016 at 2:21pm

Good job done by PPMD and other organizations. Sarepta has started recruiting for the Essence study which is for exon 45 and exon 53 skipping. It has a placebo period of 96 weeks. Which is absurd, our children will have to go through 96 weeks of IV injection to know that they were on placebo. Is PPMD working with FDA and Sarepta on this.

Comment by Shelly on September 21, 2016 at 12:16am
That's true David! Thanks Pat!
Comment by Susan Patterson on September 19, 2016 at 4:04pm
Phone number for webinar with Sarepta??
Comment by David on September 19, 2016 at 2:09pm
Every parent with boy amenable to exit 51 skip knows who to thank the most, and it's you Pat!

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