marked.bio

brain-penetrant IDH1/2 inhibitor · small molecule

vorasidenib

11 active trials, targeting IDH1/2. 2 active approvals for a biomarker on marked.bio.

Also known as AG-881, S095032, Voranigo.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
vorasidenib
FDA, 2024
grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, after surgery, age >=12 FDA approval 06/08/2024. Source
EU (EMA), 2025
predominantly non-enhancing grade 2 astrocytoma or oligodendroglioma, IDH1 R132 or IDH2 R172 mutation, after surgery only, age >=12 and >=40 kg EU marketing authorisation 17/09/2025. Source
Voranigo 		IDH1/2 mutation glioma (grade 2) Approved 2024 Approved 2025

In the FDA approval summary

INDIGO vs placebo, 331 randomised (1:1)
PFS HR 0.39 (0.27-0.56)

Trial details and side effects
Patients
residual or recurrent grade 2 IDH-mutant glioma after surgery only (NCT04164901)
Most common side effects
fatigue, headache, COVID-19, musculoskeletal pain, diarrhoea, nausea, seizure
Later trial
Time to next intervention: not reached vs 17.8 months, HR 0.26 (0.15-0.43).
Notes
FDA approval summary (2024); published median PFS 27.7 vs 11.1 months. Side effects are those in 15% or more of patients.
Source
FDA approval summary

In the EU summary (EPAR)

INDIGO vs placebo, 331 randomised (1:1)
PFS about 28 months vs about 11 months, HR 0.39 (0.27-0.56)

Trial details and side effects
Patients
residual or recurrent grade 2 IDH-mutant glioma after surgery only (NCT04164901)
Most common side effects
fatigue, headache, COVID-19, musculoskeletal pain, diarrhoea, nausea, seizure
Later trial
Time to next intervention: not reached vs 17.8 months, HR 0.26 (0.15-0.43).
Notes
EU figures from the EPAR summary; trial details and side effects from the US approval.
Source
EU summary (EPAR)

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
IDH1/2 mutation 11 14 Phase 3

Sponsors

Industry sponsors leading active biomarker-selected trials of vorasidenib.

  1. Servier 1
  2. Servier (Tianjin) Pharmaceutical 1

Trials

11 of 14 trials shown

  1. Testing Addition of an Anti-cancer Drug, Vorasidenib to Temozolomide, After Radiation for Advanced Brain Cancer

    Recruiting Phase 3 Alliance for Clinical Trials in Oncology Started 2026Primary completion 2033-05 NCT07215910

    temozolomidevorasidenib FDA EU

    STEP 0: Histologic diagnosis of astrocytoma, IDH-mutant (central nervous system \[CNS\] WHO grade 3)

    IDH1/2 mutationAstrocytoma, IDH-Mutant, Grade 3

  2. Vorasidenib Maintenance for IDH Mutant Astrocytoma

    Recruiting Phase 3 European Organisation for Research and Treatment of Cancer - EORTC Started 2026Primary completion 2035-04 NCT06809322

    vorasidenib FDA EU

    Integrated diagnosis of astrocytoma, IDH-mutant, WHO CNS5 grade 2 or 3, per local assessment

    IDH1/2 mutationIDH-mutant Grade 2 or 3 Astrocytoma

  3. Phase 3 Study of Vorasidenib (S095032/AG-881) in Asian Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 orIDH2 Mutation

    Active, not recruiting Phase 3 Servier Started 2024Primary completion 2026-05 NCT06780930

    vorasidenib FDA EU

    Have confirmed IDH1 (IDH1 R132H/C/G/S/L mutation variants tested) or IDH2 (IDH2 R172K/M/W/S/G mutation variants tested) g…

    IDH1/2 mutationResidual or Recurrent Grade 2 IDH Mutant Glioma

  4. Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

    Active, not recruiting Phase 3 Institut de Recherches Internationales Servier Started 2020Primary completion 2022-09 NCT04164901

    vorasidenib FDA EUMatching Placebo

    Have confirmed IDH1 (IDH1 R132H/C/G/S/L mutation variants tested) or IDH2 (IDH2 R172K/M/W/S/G mutation variants tested) g…

    IDH1/2 mutationGrade 2 Glioma, Residual Glioma, Recurrent Glioma

  5. Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

    Active, not recruiting Phase 1/2 Institut de Recherches Internationales Servier Started 2025Primary completion 2027-11 NCT06478212

    vorasidenib FDA EUtemozolomide

    Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

    IDH1/2 mutationIDH1-mutant Glioma, IDH2-mutant Glioma

  6. A Multicenter Safety and Immunogenicity Window-of-opportunity Phase 1 Interventional Trial Assessing the Combination of IDH1 Vaccine (IDH1-vac) and Vorasidenib in Adult Patients With Recurrent IDH1R132H-mutant Astrocytoma, Grade 2 - 4 (iVOCAL)

    Not yet recruiting Phase 1 Heidelberg University Starts 2027Primary completion 2032-12 NCT07877415

    IDH1R132H-vacvorasidenib FDA EUAldara - Imiquimod 5%

    …equivocal first, second or third recurrence of a histologically and molecularly confirmed IDH1R132H-mutated astrocytoma WHO grade 2, 3 and 4 recurrent and/or progressive after radiotherapy …

    IDH1/2 mutationRecurrent IDH1R132H-mutant Astrocytoma, Grade 2 - 4

  7. VorAsidenib With Lomustine In Patients With rEcurrent IDH-mutaNT Glioma Harboring IDH1 and/or IDH2 Mutations

    Not yet recruiting Phase 1 Megan Mantica Started 2026Primary completion 2030-06 NCT07629089

    vorasidenib FDA EULomustine

    Must have recurrent IDH-mutant oligodendroglioma or astrocytoma grade 2-4 harboring IDH1 and/or IDH2 mutation per WHO 20…

    IDH1/2 mutationRecurrent Glioma, IDH-mutant Gliomas

  8. ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas

    Recruiting Phase 1 Katy Peters, MD, PhD Started 2025Primary completion 2027-08 NCT05609994

    vorasidenib FDA EU

    IDH1R132H expression in primary tumor

    IDH1/2 mutationLow Grade Glioma of Brain

  9. Study of Vorasidenib and Pembrolizumab Combination in Recurrent or Progressive IDH-1 Mutant Glioma

    Active, not recruiting Phase 1 Institut de Recherches Internationales Servier Started 2023Primary completion 2026-02 NCT05484622

    vorasidenib FDA EUpembrolizumab

    Documented IDH1-R132H gene mutation

    IDH1/2 mutationAstrocytoma, Oligodendroglioma

  10. Vorasidenib Guided by AGX PET in Recurrent/Low-grade Glioma

    Not yet recruiting No phase Huashan Hospital Started 2026Primary completion 2027-06 NCT07469735

    vorasidenib FDA EU

    Histologically or molecularly confirmed WHO 2021 grade 2 or 3 IDH1/2-mutant diffuse glioma with recurrent or residual disease

    IDH1/2 mutationGlioma, Diffuse Glioma, Recurrent Gliomas

  11. A Multicentre, Retrospective-prospective Real-world Study: to Evaluate the Effectiveness and Safety of Vorasidenib in Patients With Isocitrate Dehydrogenase IDH1/2 Mutant Grade 2 Astrocytoma or Oligodendroglioma (VICTORIA Study)

    Active, not recruiting No phase Servier (Tianjin) Pharmaceutical Started 2025Primary completion 2028-04 NCT06969352

    vorasidenib FDA EU

    Patients with tissue-confirmed diagnoses of Grade 2 astrocytoma or oligodendroglioma with IDH1 or IDH2 mutation

    IDH1/2 mutationGliomas