Breakthrough Therapy
FDA designation that fast-tracks review of drugs with substantial improvement on clinically significant endpoints.
Definition
Breakthrough Therapy designation is granted to drug candidates that have shown preliminary clinical evidence of substantial improvement over existing therapies on at least one clinically significant endpoint. The designation includes more intensive FDA guidance and eligibility for rolling review.
See also
- Priority review — FDA designation that shortens NDA/BLA review to 6 months for drugs offering significant improvement.
- Accelerated approval — FDA pathway allowing approval based on a surrogate endpoint, requiring confirmatory post-approval trials.