FDA approves vorasidenib for IDH1/IDH2-mutant grade 2 glioma

Later-Stage HumanFDA-approved for the exact stated cancer/useNot addressed

The FDA approved vorasidenib (Voranigo) for adult and pediatric patients (12 years and older) with grade 2 astrocytoma or oligodendroglioma carrying a susceptible IDH1 or IDH2 mutation, following surgery, in patients who had not yet received chemotherapy or radiotherapy — based on the randomized, double-blind, placebo-controlled, phase 3 INDIGO trial (ClinicalTrials.gov NCT04164901; primary publication DOI 10.1056/NEJMoa2304194). INDIGO randomized 331 patients 1:1; the trial’s primary endpoint, progression-free survival by blinded independent review, was significantly longer with vorasidenib than placebo, as was time to next intervention. This is a targeted, oral therapy for a molecularly defined, treatment-naive (beyond surgery) grade 2 population only — it does not apply to grade 3 or 4 IDH-mutant glioma, or to patients who have already received chemotherapy or radiation. Elevated liver enzymes are a known risk with this drug and the label reportedly requires baseline and periodic liver-function monitoring.

What This Means

In the randomized, double-blind, placebo-controlled INDIGO trial, vorasidenib significantly delayed disease progression (by blinded independent review) and delayed the time to the next anticancer intervention, compared with placebo, in a molecularly defined, treatment-naive-beyond-surgery grade 2 IDH-mutant glioma population.

What This Doesn't Mean

This does not apply to grade 3 or 4 IDH-mutant glioma, and does not apply to patients who have already received chemotherapy or radiation for their glioma. The trial's primary endpoint was progression-free survival and time to next intervention, not overall survival — this should not be described as a proven overall-survival benefit.

Why It Matters

Offers an oral, targeted option that may let a molecularly selected group of grade 2 glioma patients delay chemotherapy or radiation therapy, and the toxicity that comes with them, for a meaningful period.

Promise & Proof

Proof — strength of the evidence 5 / 5

Base score from evidence stage: Later-Stage Human.
FDA-approved for the exact stated cancer/use.

Promise — potential significance if later evidence holds up 3 / 5

Baseline — a validly-evidenced story starts here; every further point below is an explicit, checkable reason.
The record includes substantive editorial reasoning about why this discovery matters.
A distinct follow-on/confirmatory study or trial is already cited as a secondary source — the field is already building on this finding.

These two ratings are automatically computed from this story's own structured evidence fields and are shown separately on purpose — they are never combined into one score. They describe this record, not a medical recommendation: they do not establish medical certainty, do not substitute for reading the cited sources, and are recalculated whenever the underlying evidence changes.

Population / Applicability

Studied in: Adults and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma carrying a susceptible IDH1 or IDH2 mutation, following surgery (biopsy, subtotal, or gross total resection), who had not yet received chemotherapy or radiotherapy — this does not apply to grade 3 or 4 IDH-mutant glioma, or to patients who have already received chemotherapy or radiation for their glioma. (Mixed / all ages)

Vorasidenib carries a known risk of elevated liver enzymes and the label reportedly requires baseline and periodic liver-function monitoring — this must be disclosed, not minimized. Scope is strictly grade 2, IDH1/2-mutant, post-surgery-only glioma.

Full evidence details

Study design: Randomized controlled trial
Sample size: 331

Funding & Conflicts

Sponsor and funding disclosure details were not independently confirmed in this research pass — pending human re-check.

Regulatory status by jurisdiction

United States (FDA)

Status: FDA-approved for the exact stated cancer/use
Indication: Adult and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery, including biopsy, subtotal resection, or gross total resection.
Biomarker requirement: IDH1 or IDH2 mutation
Decision date: 2024-08-06
Source: Regulator record (opens in a new tab)

Guideline positions

Guideline

Position: Not addressed
Last verified: 2026-08-13

Primary evidence supporting this story

INDIGO trial primary results: vorasidenib in IDH1/IDH2-mutant low-grade glioma (NEJM) (opens in a new tab) (Peer-reviewed paper) [Peer-reviewed] 10.1056/NEJMoa2304194 Load-bearing source DOI 10.1056/NEJMoa2304194
INDIGO trial primary results: vorasidenib in IDH1/IDH2-mutant low-grade glioma (NEJM). DOI 10.1056/NEJMoa2304194.

Additional context

INDIGO trial registry (opens in a new tab) (Clinical-trial registry) NCT04164901 NCT04164901
INDIGO trial registry. NCT04164901.

Why Should I Trust This?

1 source at Tier 2 — Peer-reviewed primary research, trial registries, regulatory documents, official conference abstracts
Last reviewed: 2026-08-13

This panel summarizes real, checkable facts about this record's own sources and review status — it is not a trust score, and reading it is not a substitute for reading the cited sources yourself.

Discovery Timeline

Every recorded change to this record's content or publication status, in order.

2026-08-13 Status change — Status changed from "Legacy / Unverified" to "In editorial review".
2026-08-13 Status change — Status changed from "In editorial review" to "Verified".

Last reviewed 2026-08-13