First glioma drug in 25 years. Initial recommendation for Europe.
Gliomas are brain tumors that develop from glial or precursor cells. There are four main categories of gliomas: astrocytomas, oligodendrogliomas, and glioblastoma multiforme, including diffuse gliomas, which occur in adults. Diffuse gliomas are the most common primary malignant brain tumors in adults and are exceptionally difficult to treat due to the lack of effective therapy.
A new drug called vorasidenib is used to treat stage II diffuse glioma with IDH1/2 (isocitrate dehydrogenase) mutations. This molecularly targeted therapy slows the progression of this particularly dangerous disease. It works by blocking IDH1/2 enzymes, which inhibits the formation of the harmful substance 2-HG (2-hydroxyglutarate), which stimulates tumor growth.
The Phase III clinical trial, called INDIGO, showed that the drug helps patients with residual or recurrent grade II glioma (with an IDH1 or IDH2 gene mutation) whose previous treatment included only surgery. The median progression-free survival (PFS) was 27.7 months for vorasidenib versus 11.1 months for a placebo (a dummy treatment).
Time to next intervention (TTNI) and the exploratory endpoint of tumor growth rate (TGR) also benefited this drug. According to calculations by the Blinded Independent Review Committee (BIRC), it reduced tumor volume by an average of 2.5% every six months, while in patients randomized to placebo, tumor volume increased by an average of 13.9% every six months.
This therapy was already approved by the Food and Drug Administration (FDA) in the United States in 2024. It has also been approved for use in Canada, Australia, Israel, the United Arab Emirates, Saudi Arabia, and Switzerland. Its approval is currently under consideration in the United Kingdom, Japan, and several other countries.
In the European Union, vorasidenib is to be used in adults and adolescents over 12 years of age and weighing at least 40 kg who have only undergone surgery and who do not require immediate radiotherapy or chemotherapy.
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