Sponsors don’t seem worried that US FDA will become skeptical of the North Star Ambulatory Assessment (NSAA) because Sarepta says it was not sensitive enough to detect patient changes in the confirmatory trial for its DMD gene therapy Elevidys.
Sarepta Therapeutics, Inc. may have unearthed new questions about a popular Duchenne muscular dystrophy endpoint during its development of the gene therapy Elevidys, but they do not appear to be cascading across gene therapy development in the rare disease.
Elevidys (delandistrogene moxeparvovec-rokl) is the first of several highly anticipated gene therapies intended to treat DMD. Sarepta is using the...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
