FDA Approval Summary: Vorasidenib for IDH -Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery
- Study type
- Other
- Study name
- FDA Approval Summary: Vorasidenib
- Design
- FDA regulatory approval summary based on a randomised, multicentre, double-blind trial of vorasidenib versus placebo
- Sample size
- 331 patients (progression-free survival assessment)
- Source
- Clinical Cancer Research · 2025
In plain words
The US Food and Drug Administration (FDA) approved the drug vorasidenib for patients aged 12 and over with certain grade 2 brain tumours (astrocytoma or oligodendroglioma) that have an IDH1 or IDH2 mutation and have been operated on. In a large trial, this drug greatly lowered the risk of the disease growing compared with a placebo (dummy pill). The most common side effects were tiredness, headache, muscle and bone pain, diarrhoea, nausea and seizures. The decision to take the drug should be made with your treating doctor.
Key results
Treatment arms and regimens
Vorasidenib (Voranigo) 40 mg orally once daily (children aged 12 years and older weighing less than 40 kg: 20 mg), until progression or unacceptable toxicity. Liver tests every 2 weeks for the first 2 months, then monthly for up to 2 years; dose reduction to 20 mg and then 10 mg.
Matching oral placebo once daily (INDIGO trial).
Source of the regimen details: FDA label for vorasidenib (VORANIGO, DailyMed; dose, liver monitoring and dose reduction)
Doses are given for the information of specialists, as in this study’s protocol, and are not a prescription. Every drug must be prescribed by the treating physician for the individual patient.
Study population
Adults and children aged 12 years and older with grade 2 astrocytoma or grade 2 oligodendroglioma with a susceptible mutation in the isocitrate dehydrogenase 1 or 2 gene (IDH1/2) after surgery, including biopsy, subtotal resection or gross total resection. Other inclusion criteria are not given in the abstract.
Endpoints
The main objective was to show the efficacy of vorasidenib based on radiographic progression-free survival (PFS) by blinded independent central review, using modified Response Assessment in Neuro-Oncology criteria for low-grade glioma (RANO-LGG).
Safety and side effects
The most common adverse reactions (incidence 15% or more) were fatigue, headache, COVID-19, musculoskeletal pain, diarrhoea, nausea and seizures. The rate of grade 3 or higher adverse events is not reported in the abstract.
Clinical significance and place in guidelines
This approval is the first FDA-approved targeted therapy for IDH-mutant grade 2 gliomas and offers a new option for patients after surgery. According to the abstract, the substantial reduction in the risk of progression or death (HR 0.39) was the basis of approval. The drug's status in clinical guidelines and approvals by other agencies (such as the EMA) should be checked separately, as they are not reported in this abstract.
Limitations
This article is a regulatory approval summary, not a full trial report. The abstract gives no data on overall survival, grade 3 or higher adverse events, or dose and schedule. The FDA considered progression-free survival an appropriate endpoint given the long natural history of this disease and the randomised design.
This summary was prepared by the editorial team from the published paper; the original paper is the final reference. Read the original abstract on PubMed.