Today’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE clinical trial. This is an important step forward for our community, providing the possibility of options for families participating in clinical trials.

Here’s a closer look at the voting:

Question 1:

Vote: Use of indwelling central venous access device in the ESSENCE clinical trial should be allowed.

• Yes – 14
• No – 0

It's a YES! In discussion, a few said they came in thinking they had thought they'd say no. After hearing the compelling testimony and presentations by families and experts, including firsthand benefit/risk stories, all committee members voted YES.

Question 2:

Following the extremely positive discussion around Question 1, the committee discussed Question 2, which was a non-voting question. Recommendations to include:

• Protocol amended as a discretionary choice (port not a requirement)
• Needs to be written so that this doesn't require protocol revisions at local IRBs – but allows flexibility on the part of local IRBs
• Decision should be left to the patient, PI, physician, and surgeon 

Next steps:

Today's Advisory Committee meeting is a strong example of how patient engagement with the FDA makes a difference. Thank you to everyone in our community who shared their experiences today. And thank you to all of the clinicians and FDA personnel who understood the urgency of this issue and provided the forum for review.

The outcome of today’s vote and discussion will be summarized into a recommendation and report for the FDA Commissioner. A final decision will be announced once the Commissioner has reviewed the summary report.