Last updated: July 16, 2026
Voranigo (vorasidenib, AG-881) is an oral, brain-penetrant, dual inhibitor of mutant IDH1 and IDH2 (Servier). It is FDA-approved (August 6, 2024) for grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery — the first targeted therapy ever approved for IDH-mutant low-grade glioma. Taken as a daily pill.
Mechanism of action:Mutations in isocitrate dehydrogenase 1 and 2 (IDH1/IDH2) make the enzyme produce an abnormal metabolite, 2-hydroxyglutarate (2-HG), which blocks tumor cells from maturing and drives cancer growth. IDH inhibitors lower 2-HG and restore normal cell differentiation — a targeted, non-cytotoxic approach.
Regulatory status:Vorasidenib: FDA Aug 6, 2024 (grade 2 IDH-mutant glioma, INDIGO Phase 3 — roughly doubled progression-free survival vs placebo, delaying chemo/radiation). The wider IDH-inhibitor class also includes FDA-approved ivosidenib (Tibsovo) for IDH1-mutant AML, cholangiocarcinoma, and MDS, and olutasidenib (Rezlidhia) for relapsed/refractory IDH1-mutant AML, plus investigational safusidenib in glioma.
For decades, a patient with a slow-growing (grade 2) IDH-mutant brain tumor faced a hard choice: watch and wait, or start chemotherapy and radiation that carry long-term cognitive costs — with nothing targeted in between. Vorasidenib changed that. It's a pill that targets the exact molecular driver of these tumors (the mutant IDH enzyme), and in the INDIGO Phase 3 trial it roughly doubled the time before the tumor progressed, delaying the need for chemo or radiation. Its August 2024 approval was a landmark — the first-ever targeted therapy for low-grade glioma, and a genuinely new option for a group of (often young) patients who previously had none.
★ Important: "IDH-mutant glioma" is NOT the same as glioblastoma
This trips up many patients and families, so it's worth being clear. Under the current WHO 2021 classification, glioblastoma is by definition IDH-wildtype (no IDH mutation). Diffuse gliomas that do carry an IDH mutation are classified separately as astrocytoma, IDH-mutant (grades 2–4) or oligodendroglioma, IDH-mutant and 1p/19q-codeleted.
So if you were told you have "glioblastoma" but your tumor testing shows an IDH1 or IDH2 mutation, under current classification you actually have an IDH-mutant astrocytoma — a distinct diagnosis with a generally better prognosis, and one for which IDH-targeted therapy is relevant. If your diagnosis and IDH status aren't clear from your pathology report, ask your neuro-oncologist — it changes which trials and treatments apply to you. See also the glioblastoma trials page.
Vorasidenib (Voranigo) — Grade 2 IDH-Mutant Astrocytoma / Oligodendroglioma (INDIGO)
Ivosidenib (Tibsovo) — IDH1-Mutant AML, Cholangiocarcinoma, MDS
Olutasidenib (Rezlidhia) — Relapsed/Refractory IDH1-Mutant AML
Curated set of recruiting / not-yet-recruiting interventional IDH-inhibitor trials in the ClinTrialFinder corpus as of July 2026 (59 total across the class), corpus-verified. IDH1/IDH2 mutation status is the gating biomarker throughout.
See the cholangiocarcinoma trials page for the full biliary IDH1 picture (ivosidenib is FDA-approved there).
IDH inhibitors are oral and generally well tolerated relative to cytotoxic chemotherapy — they do not cause the classic chemo toxicities. For vorasidenib in glioma, the most notable lab finding is elevated liver enzymes (transaminases), which requires periodic monitoring; fatigue, headache, diarrhea, and nausea are also reported. For ivosidenib and olutasidenib in myeloid disease (AML/MDS), the class carries a differentiation-syndrome warning (a potentially serious inflammatory reaction as leukemia cells mature) plus QT-interval effects. IDH1/IDH2 mutation status must be confirmed (NGS or IHC) before treatment.
What is Voranigo (vorasidenib) and how does it work?
Voranigo (vorasidenib, AG-881) is an oral, brain-penetrant dual inhibitor of mutant IDH1 and IDH2. IDH1/2 mutations produce an abnormal molecule (2-HG) that blocks tumor cells from maturing; vorasidenib lowers 2-HG and helps restore normal differentiation. A daily pill, from Servier.
What is vorasidenib FDA-approved for?
FDA-approved Aug 6, 2024 for adults and children ≥12 with grade 2 astrocytoma or oligodendroglioma carrying a susceptible IDH1/IDH2 mutation, after surgery — the first targeted therapy for IDH-mutant low-grade glioma. Based on INDIGO Phase 3 (roughly doubled progression-free survival vs placebo).
Is IDH-mutant glioma the same as glioblastoma?
No. Under WHO 2021, glioblastoma is by definition IDH-wildtype. IDH-mutant diffuse gliomas are classified separately as astrocytoma, IDH-mutant (grades 2–4) or oligodendroglioma. If you were told "glioblastoma" but your tumor is IDH-mutant, you have an IDH-mutant astrocytoma — a distinct diagnosis, generally better prognosis, and one where IDH-targeted therapy applies. Confirm with your neuro-oncologist.
What other IDH inhibitors are there besides vorasidenib?
Ivosidenib (Tibsovo) — IDH1-mutant AML, cholangiocarcinoma, MDS. Olutasidenib (Rezlidhia) — relapsed/refractory IDH1-mutant AML. Safusidenib (DS-1001) — investigational, brain-penetrant, Phase 3 in glioma (SIGMA). Vorasidenib is distinct as a dual IDH1/IDH2 inhibitor built to cross the blood-brain barrier for glioma.
How do I know if I'm eligible for an IDH-inhibitor trial?
The gating biomarker is an IDH1 or IDH2 mutation (by tumor NGS or IHC). IDH mutations occur in most grade 2–3 diffuse gliomas, ~6–10% of AML, and ~15–20% of intrahepatic cholangiocarcinoma. Trials also specify tumor type, grade, and prior treatment. If your pathology report doesn't state IDH status, ask your oncologist — it's a standard test and gates this whole class.
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