A Blow to Our Community – But Our Work Continues

Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). As stated in BioMarin’s press release, “The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.  FDA has concluded that the standard of substantial evidence of effectiveness has not been met.”

Since the Advisory Committee meeting in November, there has been a lot of speculation in our community about the FDA’s final decision. The FDA had several different response options and I think everyone in the community (myself included) was trying to guess which way they might choose to go.

We cheered for every family that participated in the Open Public Hearing portion of the Ad Comm and hoped that those stories that so clearly illustrated the benefit-risk equation would help sway the FDA to approve the drug, even if it came with conditions.  Even the FDA announcing that a decision would not be reached by their PDUFA date of December 27, created speculation and held promise because at least it wasn’t a NO.

Today’s news is a blow. Today hurts all of us…every single one. But no one is hurting more than the families who have made numerous sacrifices so that their loved ones could participate in a trial for a therapy they believe works. And while we know it doesn’t ease the pain of today’s decision, we are eternally grateful to all trial participants.

Next Steps

Our community is unfortunately used to setbacks at this point, but we will not lose our momentum because of today’s decision. There is more work to do – a lot more work to do – and PPMD has next steps already in process.

  • PPMD has reached out to BioMarin to see how we can help as they seek to find a way forward for Kyndrisa (drisapersen).

  • PPMD continues our conversations with the FDA to understand what they want to hear from patients and families in these meetings.

  • PPMD will continue to work with Sarepta and our other Duchenne partners to make sure that next week’s Ad Comm on 1/22 will be as impactful as possible and ensure the decision we all want in this community: APPROVALS.

  • But perhaps most importantly for those who have been on Kyndrisa (drisapersen), BioMarin has stated, “The ongoing Kyndrisa extension studies will continue, as will the ongoing clinical trials for other exon-skipping oligonucleotides, BMN 044, BMN 045 and BMN 053, while BioMarin is exploring next steps for this application.  Patients currently receiving Kyndrisa, BMN 044, BMN 045 and BMN 053 will remain on therapy.” PPMD and DuchenneConnect will work with patients to direct them to information regarding extension studies.

Please continue to turn to PPMD for updates on next steps for BioMarin and of course Sarepta’s Ad Comm next week.

I’m sure you are feeling a range of emotions. I know for me, I bounce between sadness and anger. But Duchenne has never been easy. And this community has never been weak. We may take a moment to mourn, to cry, to yell, but then we pick ourselves up or our neighbor helps pick us up, and we get back in there and we fight.

We fight on behalf of every person living with Duchenne. We fight for every person we have lost to Duchenne. We fight to end Duchenne.


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Comment by Jay Griffin on January 16, 2016 at 12:02pm
I must be honest, I was heartbroken when the decision came out. To all parents, we are in this fight TOGETHER. We share the SAME pain, the SAME frustrations, the SAME hope and the SAME "PROMISE".
Comment by David on January 15, 2016 at 10:42am
Kudos to Biomarin for staying the course and not letting the horrible decision making process at FDA discourage. Let's hope their investors do the same. Those on FDA panel should be haunted by their choice.
Comment by Shelly on January 14, 2016 at 12:50pm
Really painful and hard to bear but we will continue our efforts to end duchenne

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