The FDA’s 2024 approval of Voranigo (vorasidenib) marked a new era in the treatment of low-grade gliomas, the most common malignant primary brain tumors in adults. Vorasidenib is the first effective new drug developed in the last thirty years for this type of tumor. This groundbreaking targeted cancer therapy offers hope to adults and children aged 12 and older who are battling certain types of brain tumors, specifically astrocytoma or oligodendroglioma, with an isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, following surgery. Vorasidenib is the first effective new drug developed in the last thirty years to treat malignant gliomas.
The journey of Voranigo began in 2008 when Bert Vogelstein, M.D., and his team at the Johns Hopkins Kimmel Cancer Center’s Ludwig Center and colleagues from Duke University first mapped the genetic blueprint for brain cancer, the mutated IDH gene. IDH plays a crucial role in the development of low-grade gliomas. The blueprint was considered the most comprehensive genetic analysis for any tumor type, evaluating all known protein-encoding genes in brain cancer. This discovery paved the way to develop a targeted therapy that could inhibit the activity of this mutated gene, thereby slowing the growth of these previously untreatable tumors.
In June 2023, findings from a multicenter, phase 3 clinical trial of vorasidenib in 331 patients with IDH-mutant low-grade glioma published in the New England Journal of Medicine concluded that patients with grade 2 IDH-mutant glioma who received the drug had significantly improved progression-free survival. Findings continued to note that the therapy delayed the time to the next intervention compared to patients who received a placebo. Global pharmaceutical company Servier holds rights to the foundational JHU technology and sponsored the phase 3 clinical trial.
Servier secured FDA approval in August 2024, the first targeted therapy for Grade 2 IDH-mutant glioma. Voranigo specifically targets the IDH mutation, effectively slowing tumor growth and improving patient outcomes.
As Voranigo continues to make a positive impact on the lives of patients, it also sets a precedent for future innovations in targeted cancer therapies. The success of Voranigo highlights the potential for precision medicine to revolutionize the treatment of various cancers, offering more effective and personalized therapeutic options.
Voranigo Milestones
- 2008: Mutated IDH gene discovered by Bert Vogelstein, M.D., and his team at Johns Hopkins Kimmel Cancer Center’s Ludwig Center, along with colleagues from Duke University.
- June 2023: The New England Journal of Medicine published the phase 3 clinical trial data, showing significantly improved progression-free survival for patients with grade 2 IDH-mutant glioma.
- August 2024: Voranigo receives FDA approval as the first targeted therapy for Grade 2 IDH-mutant glioma.