Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm
BIOMARKER
1. Sarepta Therapeutics is preparing for an FDA advisory committee meeting to discuss its Duchenne muscular dystrophy (DMD) gene therapy, SRP-9001.
2. The discussion will focus on whether the totality of evidence supports the conclusion that SRP-9001 is reasonably likely to predict clinical benefits.
3. Evidence includes natural history, preclinical data, biomarker results, and functional results from clinical trials.
4. The panel will also address the risk-benefit analysis of SRP-9001 for treating ambulatory DMD patients.
5. Sarepta's ability to complete a confirmatory trial for the gene therapy will be assessed by the panel.
6. The biotech has a poor track record, with the confirmatory trial for its first DMD drug, Exondys 51, still unfinished.
7. Sarepta has yet to provide confirmatory clinical trial results on three drugs almost seven years after the first accelerated approval.
8. Sarepta CEO Doug Ingram believes the company's confirmatory trial, EMBARK, is in a strong position to address concerns.
9. Data from EMBARK is expected around the end of the year.
10. The FDA advisory committee meeting is scheduled for May 12.