Despite recent FDA approval, the clinical utility of vorasidenib in the treatment of IDH-mutant low-grade gliomas remains unclear. Herein, we critique the pivotal trial of vorasidenib, and highlight the questionable choice of control intervention and end points, ethical concerns, as well as the uncertain efficacy observed, and argue that the approval might be premature given the high cost of this drug and lack of clear benefit over standard treatments.
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Lazarev, S., Sindhu, K.K. Vorasidenib: a new hope or a false promise for patients with low-grade glioma?. Nat Rev Clin Oncol (2024). https://doi.org/10.1038/s41571-024-00944-5
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DOI: https://doi.org/10.1038/s41571-024-00944-5