FDA Planning Disease-Specific Guidances and Endpoints for New Gene Therapies

The FDA is planning to deliver a suite of disease-specific guidance documents next year on developing targeted gene therapies, starting with hemophilia and then more common single-gene disorders, Commissioner Scott Gottlieb told lawmakers on Capitol Hill.

“We intend to lay out modern and more efficient parameters, including new clinical measures, for the evaluation and approval of gene therapies for different high-priority diseases,” Gottlieb said during a health committee hearing on the one-year anniversary of the Senate’s passage of the 21st Century Cures Act.

The agency will provide advice on development pathways as well as possible surrogate endpoints for accelerated approvals, he continued, saying the Cures Act set the FDA on a “transformative path” in its oversight of breakthrough products.