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The New England Journal of MedicineVorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma

Vorasidenib Significantly Improved Progression-free Survival Compared to the Placebo

In a critical advancement for the treatment of IDH-mutant grade 2 gliomas, a recent double-blind, phase 3 trial has evaluated the efficacy of vorasidenib, an oral inhibitor of mutant IDH1 and IDH2 enzymes. The study focused on patients with residual or recurrent grade 2 IDH-mutant glioma who had not undergone previous treatment other than surgery, and the results shed light on the potential of vorasidenib in improving progression-free survival and delaying the time to the next intervention.

Study Design:

  • Participants: 331 patients with residual or recurrent grade 2 IDH-mutant glioma, no previous treatment other than surgery.
  • Intervention: Random assignment to receive either oral vorasidenib (40 mg once daily) or matched placebo in 28-day cycles.
  • Primary End Point: Imaging-based progression-free survival.
  • Secondary End Point: Time to the next anticancer intervention.
  • Safety Assessment: Included in the study design.

Key Findings:

  • Progression-Free Survival: Significantly improved in the vorasidenib group (median 27.7 months) compared to the placebo group (11.1 months); hazard ratio 0.39; 95% CI, 0.27 to 0.56; P<0.001.
  • Time to Next Intervention: Significantly improved in the vorasidenib group; hazard ratio, 0.26; 95% CI, 0.15 to 0.43; P<0.001.
  • Adverse Events: Grade 3 or higher in 22.8% of vorasidenib patients and 13.5% of placebo patients; increased alanine aminotransferase level of grade 3 or higher in 9.6% of vorasidenib patients.


  • Vorasidenib significantly improved progression-free survival and delayed the time to the next intervention in patients with grade 2 IDH-mutant glioma.

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“This represents the first new treatment option for low-grade diffuse glioma in more than 20 years — and the first molecularly targeted therapy specifically developed for this disease. This potential therapy could be a huge benefit to many people. Even though we call them low-grade, these tumors are far from a low-grade problem. They are incurable.”

Dr. Ingo Mellinghoff
Chair of the Department of Neurology at Memorial-Sloan
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