IDH1 inhibitorFDA-approvedInvestigational

Ivosidenib

How it works

Blocks the IDH1 enzyme, which is overactive in AML cells, disrupting cancer cell growth and survival signals.

Cancer types

Leukemia— IDH1-mutated AML

Efficacy

In clinical trials, around 40% of AML patients achieved a complete or partial response, with a median progression-free survival of approximately 5 months.

Side effects

Moderate

Side effects can be significant and may require dose adjustments or supportive medication, but the treatment is usually continued.

Evidence from research

StudyCancer typeStageEfficacy
Testing Gilteritinib with Ivosidenib or Enasidenib for Acute Myeloid LeukemiaLeukemiaphase-1—Source →
Testing Ivosidenib as Maintenance Therapy After AML TransplantLeukemiaphase-2—Source →
Testing CPX-351 and Ivosidenib for IDH1 Mutated Blood CancersLeukemiaphase-2—Source →
Testing Ivosidenib as Maintenance Therapy for Certain Blood CancersLeukemiaphase-2—Source →
New Study Examines Ivosidenib and Azacitidine for AML PatientsLeukemiaphase-3—Source →
Testing Ivosidenib and Venetoclax for IDH1 Mutated Hematologic MalignanciesLeukemiaphase-1—Source →
New Study Compares Effectiveness of Two Leukemia TreatmentsLeukemiaphase-3Treatment with OLU versus IVO was associated with significantly higher rates of complete response (RD: 0.25; 95%CI: 0.01, 0.49), transfusion independence (RD: 0.27; 95%CI: 0.01, 0.53), and OS (HR: 0.33; 95%CI: 0.11, 0.94).Source →
New Study Compares Effectiveness of Leukemia TreatmentsLeukemiameta-analysis—Source →
Lab Study Reveals How Cancer Drug Works at the Molecular LevelLeukemialab-study—Source →

This information is provided for general education only and is not medical advice. Always consult a qualified healthcare professional before making treatment decisions.