The FDA approved vorasidenib (Voranigo) on August 6, 2024 — a targeted therapy for IDH-mutant low-grade glioma, a type of brain cancer most commonly diagnosed in younger adults. The approval is specifically for patients 12 and older with grade 2 astrocytoma or oligodendroglioma carrying a susceptible IDH1 or IDH2 mutation, following surgery. The drug works by blocking the mutated IDH1/IDH2 enzymes, slowing the cancer’s growth.
The approval traces back to a 2008 discovery at the Johns Hopkins Kimmel Cancer Center, where Dr. Bert Vogelstein’s team first identified the IDH mutation in brain cancer — a finding that helped usher in the era of precision cancer medicine. In a phase 3 trial of 331 patients published in the New England Journal of Medicine, vorasidenib significantly improved progression-free survival and delayed the need for further treatment compared to placebo. For select patients who have not yet had radiation or chemotherapy, it may delay the need for those treatments — with one of the trial’s investigators calling it a potential new standard of care. (The Hopkins announcement discloses the university holds related patents and that the quoted investigator is a paid advisor to the drug’s maker, Servier.)
The most commonly reported side effects were fatigue, headache, musculoskeletal pain, diarrhea, nausea, and seizure, plus liver enzyme elevations seen on lab tests.
One important note for our community: this approval is for IDH-mutant grade 2 gliomas, not glioblastoma itself. But the IDH discovery reshaped how gliomas are classified and continues to open doors for research across other brain tumor types.
