New FDA guidance aims to accelerate personalized gene therapies for rare disease

HHS, through the FDA, issued a new draft guidance to help sponsors develop and seek approval for personalized gene therapies designed to treat patients with rare genetic diseases.

The guidance, Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management, provides a clear regulatory framework for submitting and managing BLAs for individualized genome editing products.

“By providing a clear and scientifically rigorous framework for BLA submission and post-approval management, we are helping to ensure that patients — including those with ultra-rare conditions for whom no treatment currently exists — can more efficiently access therapies that may be life-changing,” said Karim Mikhail, B. Pharm., M.S., CBER director.

Central to the framework is the concept of the Individualized Feature Space (IFS), a set of pre-defined boundaries that would allow sponsors to add new patient-specific gene-editing therapy variants to an existing BLA without additional clinical trials, provided the variants fall within predefined boundaries and laboratory data demonstrate adequate safety and activity.

The guidance also addresses manufacturing, safety testing, including risks of unintended genetic changes, and product naming and labeling. It describes how sponsors can add new patient-specific variants of an approved therapy through a streamlined review process, without requiring new clinical trials, by relying on clinical trials that supported approval of the BLA together with laboratory (in vitro) data for the new patient-specific variant.

The FDA is inviting public comment on the draft guidance.