In the phase 3 INDIGO trial, vorasidenib significantly improved progression-free survival and time to next intervention over placebo for IDH1/2-mutant glioma. The treatment reduced tumour growth rate and improved seizure control without adversely affecting quality of life or neurocognitive function.
Study
|
Randomised, double-blind, placebo-controlled, phase 3 trial [INDIGO] |
| Patients aged 12 or older with residual or recurrent grade 2 IDH1/2-mutant diffuse glioma |
| Vorasidenib (n=168) vs Placebo (n=163)
|
Efficacy
|
mPFS: Not reached vs 11.4 mos (HR 0.35) |
| Time to next intervention: Not estimated vs 20.1 mos (HR 0.25) |
| Tumour growth rate: -1.3% vs 14.4%
|
Safety
|
Grade >=3 AEs: increased alanine aminotransferase (10% vs 1%), increased aspartate aminotransferase (5% vs None), seizures (4% vs 3%) |
| Serious TEAEs: 4% vs 2% |
| No treatment-related deaths reported
|
Lancet Oncol 2025;26:1665-1675
http://doi.org/10.1016/S1470-2045(25)00472-3
Reviewed by Ulas D. Bayraktar, MD on Dec 8, 2025
