Vorasidenib shows promising results in IDH1- or IDH2-Mutant low-grade gliomas: NEJM

New research revealed that Vorasidenib, an oral brain-penetrant inhibitor of mutant IDH1 and IDH2 enzymes significantly improves progression-free survival and delayed the time to the next intervention. The trial was published in the journal The New England Journal of Medicine.

Gliomas are the most common and diffusively infiltrative central nervous system tumors that originate from the glial cells. Gliomas are classified into low-grade, atypical, and high-grade tumors based on cell morphology, mitotic activities, and molecular marker. Grade 2 gliomas with IDH mutations are malignant brain tumors that result in severe impairment and early death. In preliminary studies, Vorasidenib, an oral brain-penetrant inhibitor of mutant IDH1 and IDH2 enzymes has shown good results in IDH-mutant gliomas. Hence researchers conducted a double-blind, phase 3 trial to assess the effect of the drug on gliomas.

Patients with residual or recurrent grade 2 IDH-mutant glioma who had not undergone any previous treatment other than surgery were recruited to receive either oral vorasidenib (40 mg once daily) or matched placebo in 28-day cycles. Assessing the imaging-based progression-free survival according to blinded assessment by an independent review committee was the primary endpoint of measurement. The key secondary endpoint was the duration of the next anticancer intervention. Participants could crossover to vorasidenib from placebo based on confirmation of imaging-based disease progression. Apart from this, safety was also assessed.