This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin Phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The company was notified by the Research Institute at Nationwide Children’s Hospital (the Research Institute) that they received a letter from the FDA on July 24, 2018, stating that their micro-dystrophin gene therapy trial was placed on clinical hold due to the presence of a trace amount of DNA fragment in research-grade third-party supplied plasmid. Preliminary in-vivo testing performed by the Research Institute indicates that the trace fragment does not result in protein expression and is quickly cleared. The hold was not due to an adverse event of a patient dosed with gene therapy.
According to the community letter (below) issued by Sarepta’s President & CEO, Douglas Ingram, Sarepta understands the source of the issue and have a plan with Nationwide Children’s Hospital to keep their clinical program on track.
PPMD appreciates that the field of gene therapy is evolving swiftly. Last month, the FDA issued a series of guidances on gene therapy providing updated information as to trial standards and expectations. In short, those guidances stated that – in some instances – FDA will allow gene therapy manufacturers to utilize Phase 1/2 data as pivotal data. To that end, on a call earlier today, Doug Ingram articulated a plan for engagement with the FDA to ensure that both the plan to move forward beyond the clinical hold, as well as to move into cohort C of the clinical trial, reflected the FDA’s expectations as outlined in the guidances.
We appreciate that Sarepta is taking every opportunity to understand the implications of these guidances and the most expeditious path to approval and access for our community. We are anxious to learn more about their next steps and timeline as these conversations progress.
For more information about this clinical hold, please read the full press release here.
To learn more about this and other trials occurring in gene therapy, click here for presentations from PPMD’s 2018 Annual Conference.
Dear Duchenne Community,
This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The press release describing the clinical hold may be found here: http://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-phase-12a-duchenne-muscular.
The clinical hold resulted from an out-of-specification lot of gene therapy material. Without repeating all that is said in the release, let me say that we understand the source of the issue, have been given clear guidance on how to address it, and have a plan to keep our clinical program on track.
We are committed to holding ourselves to high standards of quality while we progress our programs with a sense of exigency. I will provide additional updates as we address the clinical hold with the goal of commencing our potential pivotal trial by the end of 2018.
Douglas S. Ingram
President and Chief Executive Officer
Sarepta Therapeutics, Inc