Dr Nagpal on the FDA Approval of Vorasidenib for Grade 2 Astrocytoma or Oligodendroglioma

Seema Nagpal, MD, clinical professor, neurology and neurological sciences, Stanford University, discusses the August 6, 2024 FDA approval of vorasidenib (Voranigo) for the treatment of adult and pediatric patients at least 12 years of age with grade 2 astrocytoma or oligodendroglioma harboring a susceptible IDH1 or IDH2 mutation.

Nagpal explains that this approval marks a significant milestone as vorasidenib is the first new medication approved for low-grade glioma in over 20 years and the first targeted therapy for this population. Vorasidenib is noteworthy for its targeted approach to treating IDH-mutant gliomas, providing a new therapeutic option for patients with these specific genetic alterations, according to Nagpal.

Nagpal highlights that the regulatory decision was based on data from the double-blind, phase 3 INDIGO trial (NCT04164901) that demonstrated a substantial improvement in progression-free survival (PFS) for patients treated with vorasidenib compared with placebo. In the trial, the median PFS was 27.7 months (95% CI, 17.0-not estimated) for patients receiving vorasidenib vs 11.1 months (95% CI, 11.0-13.7) for those receiving placebo (HR, 0.39; 95% CI, 0.27-0.56; P < .0001), illustrating a clear benefit with the targeted agent in delaying disease progression, Nagpal emphasizes.

Moreover, vorasidenib also significantly delayed time to next intervention, such as surgery, radiation, or additional chemotherapy. The median time to next intervention was not yet reached in the vorasidenib arm compared with 17.8 months in the placebo arm (HR, 0.26; 95% CI, 0.15-0.43; P < .0001), underscoring vorasidenib’s capacity to extend the period during which patients can avoid further invasive treatments, Nagpal says.

Vorasidenib’s tolerability and targeted mechanism of action make it a promising addition to the treatment paradigm for grade 2 IDH-mutant gliomas, offering a new standard of care that addresses a critical gap in treatment for this patient population, Nagpal notes.

Nagpal concludes that the introduction of vorasidenib to the glioma treatment armamentarium provides both clinicians and patients with a new and effective option that enhances disease control and potentially improves quality of life by delaying the need for further therapeutic interventions.