As Sarepta again brings a product application from the brink of rejection to the cusp of approval, the one-month review delay creates more assurance the confirmatory EMBARK trial will be completed as well as providing more time for label negotiations with the US FDA.
Sarepta Therapeutics, Inc. must wait another month for the likely accelerated approval of its proposed gene therapy for Duchenne muscular dystrophy, as well as deal with a restricted patient population, at least at the start of marketing.
The FDA needs more time to complete labeling negotiations and postmarketing discussions
The company announced on 24 May that the US Food and Drug Administration needed more time to complete its review...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
