Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
A US Food and Drug Administration advisory committee review of Sarepta Therapeutics, Inc.’s SRP-9001 (delandistrogene moxeparvovec) is expected to focus on whether the increase in dystrophin production seen with the gene therapy is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, the company said.
The decision to convene the public meeting also appears to reflect the agency’s interest in exploring the use of surrogate...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
