NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend ivosidenib (TIBSOVO®) for newly diagnoseda and R/R AML with an IDH1 mutation1
aNot eligible for or declines intensive remission induction therapy. See NCCN Guidelines® for full recommendation including age.

TIBSOVO® was studied in patients with difficult-to-treat AML1

Selected baseline demographic and disease characteristics (N=28)1
Median age (years) (min, max) 77 (64, 87)
ECOG PS
0 21%
1 57%
2 18%
3 4%
ELN risk category
Intermediate 32%
Adverse 68%
Transfusion dependent at baselinea 61%
Type of AML
De novo AML 21%
AML-MRC 68%
Therapy-related AML 11%
Prior HMA for antecedent hematological disorder 46%
aPatients were defined as transfusion dependent at baseline if they received any RBC or platelet transfusion occurring within 56 days prior to the first dose of TIBSOVO.1
  • Comorbidities that precluded the use of intensive induction chemotherapy included: baseline ECOG PS ≥2, severe cardiac or pulmonary disease, hepatic impairment with bilirubin >1.5 times the upper limit of normal, or creatinine clearance <45 mL/min1
  • 11%
    Patients who had therapy-related AML1
  • 68%
    Patients who had AML-MRC1

50% of patients had a history of MDS2
46% of patients had prior HMA therapy for an antecedent hematologic disorder1

ECOG PS, Eastern Cooperative Oncology Group Performance Status; ELN, European LeukemiaNet; HMA, hypomethylating agent; MDS, myelodysplastic syndrome; MRC, myelodysplasia-related changes.

TIBSOVO delivered strong and durable responses as an oral, single agent in difficult-to-treat disease1,2

  • 28.6% (8/28) achieved CR (95% CI, 13.2-48.7);
    14.3% (4/28) achieved CRh (95% CI, 4.0-32.7)1
58% of those who achieved CR or CRh (7/12) were in remission at 12 months after initiating treatment2
Median duration of response1,b
DOCR DOCR+CRh
NE (95% CI, 4.2-NE) NE (95% CI, 4.2-NE)
bDOCR and DOCR+CRh were defined as time since first response of CR or CR/CRh, respectively, to relapse or death, whichever is earlier.1

Median DOCR and median DOCR+CRh were not estimable (NE), with 5 patients (41.7%) who achieved CR or CRh remaining on TIBSOVO treatment (treatment duration range: 20.3-40.9 months).1

Time to response in patients who achieved CR or CRh1,2

Transfusion independence was seen in 41% of transfusion-dependent patients who received TIBSOVO1

  • 55% of patients who were transfusion independent at baseline (6/11) remained so1
  • Patients were defined as transfusion dependent at baseline if they received any RBC or platelet transfusion occurring within 56 days prior to the first dose of TIBSOVO. Patients were defined as transfusion independent if they became independent of transfusions during any 56-day postbaseline period1
RBC, red blood cell.

Reference: 1. TIBSOVO [package insert]. Cambridge, MA: Agios Pharmaceuticals, Inc.; 2019. 2. Data on file. Agios Pharmaceuticals, Inc.
 

TIBSOVO® was studied as a single agent in both the newly diagnosed and R/R AML settings1

TIBSOVO is a first-in-class agent that inhibits the mutant IDH1 enzyme to induce myeloid differentiation1

  • The pivotal trial for TIBSOVO was an open-label, single-arm, multicenter trial1
  • IDH1 mutations were identified by a local or central diagnostic test and confirmed retrospectively using the Abbott RealTimeTM IDH1 assay, which is the FDA-approved test for selection of patients with AML for treatment with TIBSOVO1
Patients were assigned a starting dose of TIBSOVO 500 mg daily and received treatment until disease progression, development of unacceptable toxicity, or undergoing hematopoietic stem cell transplantation1
  • 28 IC-ineligible patients with newly diagnosed AML were evaluated for safety and efficacy
  • 179 patients were evaluated for safety and 174 for efficacy in the R/R AML population

Efficacy was established based on the rate of CR and/or CRh, duration of CR+CRh, as well as on the rate of conversion from transfusion dependence to transfusion independence1

CR, complete remission, defined as <5% blasts in the bone marrow, no evidence of disease, and full recovery of peripheral blood counts (platelets >100,000/microliter and absolute neutrophil counts >1000/microliter); CRh, complete remission with partial hematological recovery, defined as <5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/microliter and absolute neutrophil counts >500/microliter); IC, intensive chemotherapy; R/R, relapsed or refractory.1

Reference: 1. TIBSOVO [package insert]. Cambridge, MA: Agios Pharmaceuticals, Inc.; 2019.
 

INDICATIONS

TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

  • Adult patients with newly diagnosed AML who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Indications & Important Safety Information

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

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