FDA proposes fast-tracking gene therapies for rare diseases with strong scientific basis.

The U.S. FDA has proposed a new regulatory pathway to fast-track approval of customized gene therapies for rare diseases, allowing approval based on a plausible biological mechanism and evidence of targeting the disease’s root cause, even with limited patient data. The move aims to accelerate access to treatments like CRISPR-based therapies for ultra-rare conditions where traditional large-scale trials are impractical. It enables commercialization of approved therapies—unlike current compassionate use rules—and follows recent changes to reduce standard trial requirements. The draft guidance, open for public comment for 60 days, applies only to well-understood genetic disorders with strong scientific rationale and is not intended for common diseases.