Sarepta Therapeutics, Inc.
NEWS
The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
Gene therapy company Sarepta Therapeutics provided additional data for SRP-5051 in treating Duchenne muscular dystrophy patients who are amenable to exon 51 skipping.
Despite failing to hit the primary endpoint in the Phase III EMBARK study, the company plans to file for a label expansion for its Duchenne muscular dystrophy gene therapy Elevidys.
Recent FDA approvals of novel drugs based on less-than-stellar clinical evidence point to a trend toward regulatory flexibility—particularly in indications with very high unmet need.
Recent drug approvals have shone a light on the role that patient advocacy groups can play in the regulatory process—but some experts have questions about the ethics of this influence.
The FDA’s CBER approved nine novel therapies in the first half of 2023, with several more high-profile decisions on deck for the second half of the year.
Shares of Sarepta dropped 11% a day after securing accelerated approval for the first gene therapy to treat Duchenne muscular dystrophy over concerns about the potential for label expansion.
The FDA has four target action dates this week, including ones for Argenx’s subcutaneous efgartigimod in generalized myasthenia gravis and Sarepta’s gene therapy for Duchenne muscular dystrophy.
The extensive discussion between the FDA’s advisory committee, company representatives and other interested parties could serve as a cautionary tale to developers of future gene therapies.
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