Same Trial, Different Endpoints: FDA Guided Idhifa To Full Approval By Recasting Clinical Analyses

US FDA conducted extensive re-analyses of Celgene's AML therapy Idhifa, resulting in full approval based on complete remission findings instead of the accelerated approval based on overall response rate sought by the NDA. 

In an unusual turn of events, The US FDA's disagreement with the primary endpoint used in the pivotal (and only) clinical study supporting Celgene Corp.'s NDA for the acute myeloid leukemia (AML) therapy Idhifa resulted in a better approval outcome than the company had anticipated.

Review documents for Idhifa (enasidenib) describe how an NDA seeking accelerated approval based on overall response rate (ORR) became a...

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