IDHIFA Clinical Trial Design

Approval of IDHIFA was based on results from a

IDHIFA was evaluated in an open-label, single-arm, multicenter, 2 cohort clinical trial of 199 adult patients with R/R AML and an IDH2 mutation. Patients' IDH2 mutations were identified by a local diagnostic test and retrospectively confirmed by or prospectively identified by the Abbott RealTime™ IDH2 assay. IDHIFA was given orally at a starting dose of 100 mg daily until disease progression or unacceptable toxicity. Dose reductions were allowed to manage side effects. Patients had a median age of 68 years (range, 19 to 100) and received a median of 2 prior anticancer regimens (range, 1 to 6). More than half (52%) were refractory to previous therapy.

  • Efficacy was established on the basis of the rate of complete response (CR)/complete response with partial hematologic recovery (CRh), the duration of CR/CRh, and the rate of conversion from transfusion dependence to transfusion independence
  • The median follow-up was 6.6 months (range, 0.4 to 27.7 months)

Patient Demographics

Demographic and disease characteristics


Demographic and disease characteristics
R/R AML, N=199

100-mg daily dose

Median age (years)
  • 68 (range, 19-100)
Age categories, n (%)
<65 years76 (38)
≥65 years to <75 years74 (37)
≥75 years49 (25)
Sex, n (%)
Male103 (52)
Female96 (48)
Race, n (%)
White153 (77)
Black10 (5)
Asian1 (1)
Native Hawaiian/other Pacific Islander1 (1)
Other/not provided34 (17)
ECOG PSa, n (%)
046 (23)
1124 (62)
228 (14)
Relapsed AML, n (%)95 (48)
Refractory AML, n (%)104 (52)
IDH2 mutationb, n (%)
R140155 (78)
R17244 (22)
Median time from initial AML diagnosis
(months) (172 patients)
11.3 (range, 1.2-129.1)
Cytogenetic risk status, n (%)
Intermediate98 (49)
Poor54 (27)
Missing/failure47 (24)
Prior stem cell transplants for AML, n (%)25 (13)
  • Transfusion dependent at baselinec, n (%)
  • 157 (79)
Number of prior anticancer regimensd, n (%)
189 (45)
264 (32)
≥346 (23)
Median number of prior therapies2 (range, 1-6)

a1 patient had missing baseline ECOG PS.

b For 3 patients with different mutations detected in bone marrow compared to blood, the result of blood is reported.

c Patients were defined as transfusion dependent at baseline if they received any RBC or platelet transfusions within the 8-week baseline period.

d Includes intensive and/or nonintensive therapies.

ECOG PS, Eastern Cooperative Oncology Group performance status; RBC, red blood cell.

IDHIFA Clinical Trial Results

IDHIFA efficacy outcomes


n (%)37 (19%)
(95% CI, 13%-25%)
9 (4%)
(95% CI, 2%-8%)
46 (23%)
(95% CI, 18%-30%)
Median DORc (months)8.2
(95% CI, 4.7-19.4)
(95% CI, 0.7-NA)
(95% CI, 4.3-19.4)
 n (%)Median DORc (months)
CRa37 (19%)
(95% CI, 13%-25%)
(95% CI, 4.7-19.4)
CRhb9 (4%)
(95% CI, 2%-8%)
(95% CI, 0.7-NA)
CR/CRh46 (23%)
(95% CI, 18%-30%)
(95% CI, 4.3-19.4)

aCR was defined as <5% of blasts in the bone marrow, no evidence of disease, and full recovery of peripheral blood counts (platelets >100,000/μL and ANC >1,000/μL).

b CRh was defined as <5% of blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/μL and ANC >500/ μL).

c DOR was defined as time since first response in CR or CRh to relapse or death, whichever is earlier.

ANC, absolute neutrophil counts; CI, confidence interval; DOR, duration of response; NA, not available.


 Time to first response
Time to best response of CR/CRh
Patients achieving CR/CRh (n=46/199)1.9
(range, 0.5-7.5)
(range, 0.6-11.2)

Of the 46 patients who achieved a best response of CR/CRh, 39 (85%) did so within 6 months of initiating IDHIFA

rbc- and platelet-transfusion independence

Among the 157 patients who were dependent on RBC and/or platelet transfusions at baseline, 53 (34%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.

Of the 42 patients who were independent of both RBC and platelet transfusions at baseline, 32 (76%) remained transfusion independent during any 56-day post-baseline period.


IDHIFA® (enasidenib) is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.

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Important Safety Information


Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.



Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 14% of patients treated with IDHIFA experienced differentiation syndrome. Differentiation syndrome has been observed with and without concomitant hyperleukocytosis, as early as 10 days and at up to 5 months after IDHIFA initiation. If differentiation syndrome is suspected, initiate systemic corticosteroids and hemodynamic monitoring until improvement. Taper corticosteroids only after resolution of symptoms. Differentiation syndrome symptoms may recur with premature discontinuation of corticosteroids. If severe pulmonary symptoms requiring intubation or ventilator support and/or renal dysfunction persist for more than 48 hours after initiation of corticosteroids, interrupt IDHIFA until signs and symptoms are no longer severe. Hospitalization for close observation and monitoring of patients with pulmonary and/or renal manifestation is recommended.

Embryo-Fetal Toxicity: Based on animal embryo-fetal toxicity studies, IDHIFA can cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with IDHIFA and for at least 1 month after the last dose. Pregnant women, patients becoming pregnant while receiving IDHIFA, or male patients with pregnant female partners should be apprised of the potential risk to the fetus.


  • The most common adverse reactions (≥20%) included total bilirubin increased (81%), calcium decreased (74%), nausea (50%), diarrhea (43%), potassium decreased (41%), vomiting (34%), decreased appetite (34%), and phosphorus decreased (27%)
  • The most frequently reported ≥Grade 3 adverse reactions (≥5%) included total bilirubin increased (15%), potassium decreased (15%), phosphorus decreased (8%), calcium decreased (8%), diarrhea (8%), differentiation syndrome (7%), non-infectious leukocytosis (6%), tumor lysis syndrome (6%), and nausea (5%)
  • Serious adverse reactions were reported in 77.1% of patients. The most frequent serious adverse reactions (≥2%) were leukocytosis (10%), diarrhea (6%), nausea (5%), vomiting (3%), decreased appetite (3%), tumor lysis syndrome (5%), and differentiation syndrome (8%). Differentiation syndrome events characterized as serious included pyrexia, renal failure acute, hypoxia, respiratory failure, and multi-organ failure


Many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed infants, advise women not to breastfeed during treatment with IDHIFA and for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING.

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