# Vorasidenib

Source: https://onco.cc/drugs/vorasidenib/  
OnCo record `vorasidenib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The first targeted therapy for low-grade brain tumours, delaying the need for radiation and chemotherapy by years.

## Summary

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.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-04
- Brand: Voranigo
- Modality: Small-molecule IDH1/2 inhibitor
- Mechanism: Brain-penetrant dual IDH1/IDH2 inhibitor reducing 2-HG.
- Approvals: 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
- Dosing: Oral; 40 mg once daily (≥40 kg); 20 mg once daily (<40 kg)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Vorasidenib
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Vorasidenib

## Connected records

- biomarkers: [1p/19q codeletion](https://onco.cc/biomarkers/1p19q-codeletion-readout/), [IDH1 R132 mutation](https://onco.cc/biomarkers/idh1-r132/), [IDH2 mutation (R140 and R172)](https://onco.cc/biomarkers/idh2-mutation/)
- cancers: [Astrocytoma, IDH-mutant (grades 2 to 4)](https://onco.cc/cancers/idh-mutant-astrocytoma/), [Brain and spinal cord tumours (all types)](https://onco.cc/cancers/brain-tumours/), [Glioma & glioblastoma](https://onco.cc/cancers/glioblastoma/), [Oligodendroglioma, IDH-mutant and 1p/19q-codeleted](https://onco.cc/cancers/oligodendroglioma/)
- technologies: [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/), [IDH inhibitors](https://onco.cc/technologies/idh-inhibitors/)
- targets: [IDH1 / IDH2](https://onco.cc/targets/idh/)
- companies: [Servier](https://onco.cc/companies/servier/)
- trials: [INDIGO](https://onco.cc/trials/indigo/)
- pathways: [Cancer metabolism](https://onco.cc/pathways/cancer-metabolism/), [Epigenetic reprogramming](https://onco.cc/pathways/epigenetic-reprogramming/), [Glioma (KEGG map)](https://onco.cc/pathways/glioma-signalling/), [Glutamine addiction](https://onco.cc/pathways/glutamine-metabolism/), [Mutant IDH / 2-hydroxyglutarate](https://onco.cc/pathways/idh-2hg/)
- people: [Ingo K. Mellinghoff](https://onco.cc/people/ingo-mellinghoff/), [Patrick Y. Wen](https://onco.cc/people/patrick-wen/)
- bottlenecks: [The brain: barrier and sanctuary](https://onco.cc/bottlenecks/b-brain-delivery/)
- collections: [National Brain Tumor Society (NBTS)](https://onco.cc/collections/national-brain-tumor-society/)
- key papers: [INDIGO: vorasidenib, the first targeted drug for IDH-mutant low-grade glioma](https://onco.cc/key-papers/paper-indigo-nejm-2023/)
- terms: [1p/19q codeletion](https://onco.cc/terms/1p19q-codeletion/), [Epigenetic progenitor theory: cancer without a first mutation](https://onco.cc/terms/epigenetic-progenitor-theory/), [Metabolic theory of cancer: from Warburg to oncometabolites](https://onco.cc/terms/metabolic-theory-of-cancer/)
- roadmaps: [Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome](https://onco.cc/roadmaps/epigenetics-roadmap/), [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

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