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FDA Makes Determination Regarding In-Dwelling Ports

Time and again in our Duchenne community, we see brave kids and their warrior parents work to change the landscape. After all, it’s that exact spirit upon which PPMD was founded. And with that same spirit that family after family has transformed their personal moments into broader movements that have benefited us all.

 

On May 18, the FDA Pediatric Advisory Committee convened to determine whether to allow for the protocol of Sarepta’s ESSENCE trial to be revised to allow for the placement of in-dwelling ports. Today we are so pleased to announce that the FDA has made a determination on the referral of the ESSENCE protocol for review under 21 CFR 50.54 and sent the FDA Determination Memorandum to both UCLA and Sarepta Therapeutics. According to the determination, all conditions have been met and the protocol "may proceed with the inclusion of a totally implantable central venous access device (TICVAD)."

 

What Happens Next?

The Local IRB for each institution participating as a site in the ESSENCE study will now have the opportunity to review their trial protocol to determine whether to allow for the consideration of in-dwelling port placements within the ESSENCE trial protocol at their individual site. Participating trial site protocols will not automatically be revised based on the FDA Determination.

 

The Duchenne Community Is Grateful

We are grateful to the FDA for understanding the importance and urgency of this issue and for giving it the attention it deserves. We are grateful to the Bullers and each family who worked to raise this issue, who traveled to White Oak to testify, and who gave voice to the issue by submitting written testimony. The discussion of the Pediatric Advisory Committee on May 18th reflected that our community’s experiences were heard and considered thoroughly. The testimony made a lasting impression on the Committee.

 

We are grateful to the FDA and the members of the Pediatric Advisory Committee. We believe that the determination of this Pediatric Advisory Committee to allow for the protocol to be amended such that IRBs can provide a space for patients and providers to make the best decisions for each trial participant, is an outcome that allows for greater personal/provider choice. And we appreciate that the FDA understood the urgency and importance of this matter.

 

We are grateful to the Jett Foundation, our Duchenne partners who worked alongside us to help facilitate our community’s engagement in the Advisory Committee meeting last week.

 

We are grateful to the providers who gave voice to this issue — especially the UCLA team led by Dr. Perry Shieh and the UCLA IRB — for carving a path forward to the FDA.

 

But most of all, and at the center of it all, we are grateful to our community. The families who fought so hard to raise this issue up and the kids who’ve endured despite great discomfort.

More information

To read the full FDA determination, click here.

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