Elevidys And The EMBARK Trial: Is Another Advisory Committee Meeting Coming?
Sarepta says another public meeting is not necessary to expand the label for its DMD gene therapy, but the missed primary endpoint may require input from experts outside the FDA, and the situation renews the spotlight on CBER Director Peter Marks and his initial approval decision.
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Julie Tierney will become CBER’s deputy director for strategy, policy and legislation, a newly created position.
Oncology Center of Excellence Director Richard Pazdur also says that he will not grant an accelerated approval if the confirmatory trial is not underway, even if the product shows safety and efficacy.
Strong demand from physicians and families of boys with Duchenne has seen a rapid take-off in sales of the $3.2m gene therapy – but the FDA will have the final say on the product's future after a failed Phase III study.