Accelerated Approval
An FDA pathway allowing earlier approval based on a surrogate endpoint reasonably likely to predict clinical benefit. Companies must conduct confirmatory post-marketing trials. If confirmatory trials fail, the FDA can withdraw the accelerated approval. Elamipretide received accelerated approval for Barth syndrome.
Technical Context
Accelerated Approval under 21 CFR 314 Subpart H (drugs) or 21 CFR 601 Subpart E (biologics) allows approval based on a surrogate endpoint reasonably likely to predict clinical benefit, or on an intermediate clinical endpoint that can be measured earlier than irreversible morbidity/mortality. Post-marketing confirmatory trials (required by law since the Accelerated Approval Reform provisions of FDORA 2022) must verify clinical benefit — failure can result in expedited withdrawal proceedings. Recent FDA actions have withdrawn several accelerated approvals where confirmatory trials failed. Elamipretide received accelerated approval for Barth syndrome based on improvements in a functional endpoint. The balance between earlier patient access and uncertainty about clinical benefit is the fundamental tension of the accelerated approval pathway.