OnCo
drugsProductApproved2017

Enasidenib

Enasidenib is the IDH2 counterpart of ivosidenib, approved in the US for relapsed disease but never approved in Europe after it failed to extend survival in a confirmatory trial.

Approved 2017 (US) for relapsed/refractory IDH2-mutated AML on a single-arm study (CR+CRh ~23%). The phase 3 IDHENTIFY trial did not improve OS versus conventional care; the EMA application was withdrawn (2019), and BMS later announced market withdrawal in some territories while maintaining US availability. Illustrates the gap between response rate and survival endpoints in relapsed AML.

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Enasidenib
PubChem
Mechanism, step by step
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1.Mutant IDH2 produces 2-HG in mitochondria

Modality
Small-molecule IDH2 inhibitor
Mechanism
Allosteric inhibitor of mutant IDH2 (R140Q and R172K), lowering 2-HG and inducing differentiation.
Brand / code
Idhifa
Dosing & schedule
Route
Oral
Schedule
100 mg once daily until progression (minimum 6 months)
Monitoring
Differentiation syndrome (boxed warning), bilirubin (indirect hyperbilirubinaemia from UGT1A1 inhibition)

Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.

Medicare
Part D (self-administered)

Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).

Commercial insurance
covered with prior authorisation

Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. IDH2 mutation by an approved test.

Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.

Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

Not appraisedSearch NICE
Notes
EU marketing authorisation application withdrawn 2019; no UK licence.
NHS England
Not routinely funded

Sources: NICE search: enasidenib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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  1. 1 Aug 2017ApprovalUS

    Single-arm AG221-C-001

  2. 1 Dec 2019WithdrawalEU

    Marketing application withdrawn after EMA objections on efficacy source

Approvals

RegionYearIndication
US2017Relapsed/refractory IDH2-mutated AML

Safety

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Toxicity profile
Adverse eventAny gradeGrade 3+
Differentiation syndrome
14%
Indirect hyperbilirubinaemia
81%
Nausea
50%

Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
28 studies11 recruiting25 Phase 24 Phase 3
Search “Enasidenib” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Enasidenib
intervention: Enasidenib
Open on ClinicalTrials.gov →

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Latest papers

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Literature trend24 papers in the last 12 months-17% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Enasidenib" OR ABSTRACT:"Enasidenib" OR TITLE:"Idhifa" OR ABSTRACT:"Idhifa") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Enasidenib, not a curated reading list.

Connected

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