Spotlight On High Doses Again After Pfizer Duchenne Gene Therapy Death
US Regulator May Call For Limits
The death in Pfizer’s Phase I study could reflect wider safety issues for AAV-based gene therapy and could spur intervention from regulators in 2022.
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The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.
The company is seeking FDA accelerated approval of the closely watched gene therapy based on a surrogate marker and has proposed its fully enrolled Phase III trial to serve as a confirmatory study.
Sarepta has experience pursuing accelerated approvals in the US for Duchenne muscular dystrophy therapies, but with a costly gene therapy will payers require data from a controlled Phase III study?