publication date: Aug. 4, 2017
Drugs and Targets
Enasidenib, companion diagnostic get FDA approval for relapsed or refractory AML
FDA granted a regular approval to enasidenib (IDHIFA) for the treatment of adult patients with relapsed/refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 mutation as detected by an FDA-approved test.
The drug was initially discovered and developed by Agios Pharmaceuticals. It is sponsored by Celgene Corp.
FDA previously granted Orphan Drug and Fast Track designations as well as priority review to enasidenib.
This is the first FDA approval for relapsed/refractory AML specifically with an IDH2 mutation. FDA concurrently approved a companion diagnostic, the RealTime IDH2 Assay, used to detect the IDH2 mutation.
The enasidenib approval was based on Study AG221-C-001 (NCT01915498), an open-label, single-arm, multicenter, clinical trial of enasidenib that included 199 adults with relapsed or refractory AML who had an IDH2 mutation as detected by the RealTime IDH2 Assay.
Patients were treated with enasidenib 100 mg orally daily. Complete response and complete response with partial hematologic recovery rates, CR/CRh duration, and conversion from transfusion dependence to transfusion independence were the basis of approval.
After a median follow-up time of 6.6 months, 23% of patients experienced CR or CRh lasting a median of 8.2 months, with 19% of patients having a CR lasting a median 8.2 months, and 4% with a CRh lasting a median 9.6 months. The median time-to-first response was 1.9 months (range, 0.5 to 7.5 months) and the median time-to-best response of CR/CRh was 3.7 months (range, 0.6 to 11.2 months).
Of the 157 patients who required transfusions at the initiation of the trial, 34% no longer required transfusions during at least one 56-day time period on enasidenib. Of the 42 patients who did not require transfusions at the start of the trial, 76% maintained transfusion independence.
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