Review of The Pediatric Advisory Committee Meeting to Evaluate Ports in ESSENCE

On Thursday, May 22, 2017, The Pediatric Advisory Committee met to evaluate the use of indwelling central access devices in Sarepta Therapeutics ESSENCE study of exons-45 and 53 skipping compounds.

After listening to the principle investigator Dr. Perry Shieh from UCLA, Sarepta, the Bullers' family, the Office of Pediatric Therapeutics, and the patient and family testimony during the Open Public Hearing (OPH), the panel voted unanimously, 14-0, that the use of indwelling central venous access devices in the ESSENCE study should be allowed.

The panel also recommended to the agency that the use of indwelling central access devices remain discretionary and that the choice be made on an individual basis by the family, principle investigator, and the surgeon.

This was a very positive recommendation that will benefit many boys involved in the ESSENCE study who suffer from peripheral access difficulties during their weekly infusions.

The panel was clearly moved by the Bullers’ 20-minute presentation on the perspective of a patient involved in the ESSENCE study who experiences severe, and debilitating peripheral access issues during his weekly infusions. The panelists were educated on the difficulties of access by the OPH testimonies of additional patients and families involved in ESSENCE, and those who have experience with indwelling central venous devices in other clinical trials.

Jett Foundation is pleased that the patient perspective was taken into consideration by the panel.

Our organization, and on behalf of those affected by this positive decision, would like to thank the Bullers' family for their participation in the Advisory Committee Meeting and the ESSENCE study, as well as the other families involved in ESSENCE who testified during OPH.

We also want to thank Dr. Perry Shieh (UCLA) for his participation and commitment to his patients, and Office of Pediatric Therapeutics and Dr. Skip Nelson (FDA) for their rapid response to this serious issue.

Lastly, we thank Parent Project Muscular Dystrophy for their work to ensure that Thursday ran smoothly. We appreciate our partnership and our ability to work together for the betterment of this patient community.

The outcome of Thursday’s vote and discussion will be forwarded to the FDA Commissioner, Dr. Scott Gotlieb. Dr. Gottlieb will make the final decision on the protocol change.

We hope that Dr. Gottlieb comes to a positive decision quickly, ensuring that ESSENCE patients whot need a port to continue with the study will be able to have one as soon as possible.