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The first targeted therapy for low-grade brain tumours, delaying the need for radiation and chemotherapy by years.
Vorasidenib is a brain-penetrant dual inhibitor of mutant IDH1 and IDH2 that lowers the oncometabolite 2-hydroxyglutarate (2-HG), which drives the epigenetic block behind IDH-mutant glioma. In INDIGO it extended progression-free survival to 27.7 versus 11.1 months in grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, and it was approved in August 2024 for patients aged 12 and over, the first targeted therapy for low-grade brain tumours. Servier markets it; the dose is 40 mg once daily (20 mg below 40 kg), and raised ALT and AST are the main laboratory findings. Its purpose is to delay radiotherapy and chemotherapy and their long-term cognitive cost by years; whether it improves overall survival, and its role in higher-grade IDH-mutant tumours, are open. For a newcomer: a pill that holds slow-growing brain tumours in check and postpones harsher treatment.
Brain-penetrant dual IDH1/IDH2 inhibitor reducing 2-HG. Connects to IDH1 / IDH2.
1.Brain-penetrant drug crosses the blood-brain barrier
Background: ADC sequencing, Antigen escape (antigen loss, lineage switch), BCG-unresponsive, Castration-resistant prostate cancer (CRPC), Circulating tumour DNA (ctDNA). Also on OnCo: Resistance: how tumours escape each drug class · Lines of therapy.
Source: www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications. Doses are for orientation; the current label governs.
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. IDH1/2-mutant grade 2 glioma after surgery, per the label.
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
Sources: NICE TA1147. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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Breakthrough Therapy designation source
Grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, age ≥12 (INDIGO): first targeted therapy for low-grade glioma source
EMA approval source
| Region | Year | Indication |
|---|---|---|
| US | 2024 | Grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, age ≥12 |
| EU | 2025 | Grade 2 IDH-mutant glioma ≥12 yrs; 17 Sep 2025 |
| Adverse event |
|---|
| ALT increased |
| AST increased |
| Fatigue |
| Headache |
| COVID-19 |
| Musculoskeletal pain |
INDIGO: ~39% any grade, ~9.6% grade 3-4. Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.
| Country | Reimbursement | List price | Assistance |
|---|---|---|---|
| United States | Medicare Part D (oral); commercial plans per formulary, often with prior authorisation | not disclosed | - |
| United Kingdom | NICE: recommended for grade 2 IDH-mutant glioma after surgery (2025) | not disclosed | - |
List prices are manufacturer or Medicare figures where publicly disclosed; net prices after rebates are usually lower. Reimbursement changes; check the payer.
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Query for this drug: (TITLE:"Vorasidenib" OR ABSTRACT:"Vorasidenib" OR TITLE:"Voranigo" OR ABSTRACT:"Voranigo") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Vorasidenib, not a curated reading list.
Shares Mutant IDH / 2-hydroxyglutarate, IDH1 R132 mutation, IDH inhibitors, Astrocytoma, IDH-mutant (grades 2 to 4).
Shares Mutant IDH / 2-hydroxyglutarate, IDH1 R132 mutation, IDH inhibitors, Glutamine addiction.
Shares IDH2 mutation (R140 and R172), Mutant IDH / 2-hydroxyglutarate, IDH inhibitors, IDH1 / IDH2.
Shares IDH inhibitors, Servier, IDH1 / IDH2, Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome.
Shares National Brain Tumor Society (NBTS), 1p/19q codeletion, Astrocytoma, IDH-mutant (grades 2 to 4), Epigenetic reprogramming.
Shares 1p/19q codeletion, IDH1 R132 mutation, Astrocytoma, IDH-mutant (grades 2 to 4), Glioma & glioblastoma.
Shares Glutamine addiction, Metabolic theory of cancer: from Warburg to oncometabolites, Cancer metabolism, IDH1 / IDH2.
Shares IDH inhibitors, IDH1 / IDH2, Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET).