Sarepta Pursues DMD Gene Therapy Accelerated Approval To Be First To Market
New Data Analyses Swayed US FDA To Allow Early Filing
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?
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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.
Santhera is bouncing back after a Duchenne drug failure two years ago with idebenone, and is looking to gain approval of vamorolone in this indication as well as the much rarer Becker muscular dystrophy.
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.