Established efficacy endpoints may not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control groups and use of sham injections.
The US FDA’s clinical development advice for gene therapies to treat retinal disorders openly embraces the type of novel endpoint development that Spark Therapeutics Inc. pursued with Luxturna, the first gene therapy approved in this space.
The draft guidance includes examples of established efficacy endpoints but also encourages sponsors to develop and propose novel endpoints to...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
