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Enasidenib fails OS endpoint in confirmatory phase 3 study

Enasidenib was approved in 2017 based on phase 1/2 data. For its approval, FDA introduced CRh (CR with incomplete hematological recovery) as a new measure of activity, as well as the transfusion-dependence conversion rate.

IDHENTIFY was initiated as a confirmatory study following the anticipated accelerated approval. Unexpectedly, enasidenib received FULL approval right away. Failure of IDHENTIFY, which compared single agent enasidenib to conventional care, is unlikely to make an impact on the approval status.

BMS, which acquired enasidenib as part of the acquisition of Celgene, is not pursuing any additional studies.
An ongoing phase 3 study in tx-naïve AML is sponsored by HOVON.

AG221-C-001: NCT01915498
HOVON150AML: NCT03839771
Stein et al., Blood 2017 (PMID: 28588020)