Targeting IDH-Mutant Glioma with Vorasidenib : A New Era in Precision Neuro-Oncology.

Abstract

Advancement of molecular targeted therapies has revolutionized the treatment of several challenging and previously refractory tumors. Since the introduction of rituximab in 1997, the field of molecular targeted agents have continued to evolve. Notably, vorasidenib, a brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase (IDH) 1 and 2, has recently demonstrated promising results in the 2023 INDIGO trials with improved progression-free survival (PFS) and delayed need for subsequent interventions in patients with IDH-mutant low-grade gliomas. Although therapeutic options for glioma have long remained largely limited to surgery, radiation, and conventional chemotherapy despite extensive and ongoing research, the advent of vorasidenib opens a new chapter in glioma treatment. This review summarizes the key clinical studies of vorasidenib and discusses its potential role, limitations, and future directions in the evolving treatment paradigm of IDH-mutant gliomas.