The EMBARK trial’s availability to more patients likely contributed to the much quicker enrollment than confirmatory trials for Exondys 51, Vyondys 53 and Amondys 45, which also saw enrollment targets and eligibility ages change over the years.
The clinical trial testing Sarepta Therapeutics, Inc.’s gene therapy candidate in Duchenne muscular dystrophy enrolled remarkably faster than the company’s studies looking at three drugs that treat the rare disease, a likely function of its potential and a concern for the others’ futures.
SRP-9001 (delandistrogene moxeparvovec) is under priority review at the US Food and Drug Administration for accelerated approval. The EMBARK trial...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
