Being diagnosed with a brain tumor in your 30s or 40s—when you are building your career, raising children, and staying socially active—can upend your life. Low-grade glioma is considered a relatively slow-growing brain tumor, but it is difficult to cure completely and can progress over time into a more aggressive, high-grade glioma. Many patients experience seizures, and even after treatment they live with anxiety about recurrence and worsening disease.
Until now, treatment for low-grade glioma has largely relied on surgery, radiation therapy, and chemotherapy. However, it is often difficult to remove a brain tumor completely, and radiation and chemotherapy can have limitations because they may affect quality of life, including cognitive decline. Against this backdrop, expectations are rising that the treatment paradigm could shift with the emergence of a new targeted therapy in glioma for the first time in about 20 years.
Global pharmaceutical company Servier held a press briefing on the 12th to announce the South Korean launch of ‘Voranigo’ (generic name: vorasidenib), the first targeted therapy for IDH-mutant glioma. At the briefing, Prof. Jong-hee Jang of the Department of Neurosurgery at Yonsei University Severance Hospital and Prof. Jae-yong Kim of the Department of Neurosurgery at Seoul National University Bundang Hospital discussed the current treatment landscape for low-grade glioma and the clinical significance of the new therapy.
Voranigo received approval from South Korea’s Ministry of Food and Drug Safety in January 2026. The approved indication includes pediatric and adult patients aged 12 years and older who weigh at least 40 kg and require treatment after surgery, including biopsy, subtotal resection, or gross total resection. Specifically, it is indicated for patients with grade 2 astrocytoma or oligodendroglioma with an IDH1 or IDH2 mutation. Voranigo has also been approved in more than 40 countries worldwide, including the United States, Canada, the United Kingdom, and Japan, and is being used in real-world clinical practice.
“IDH mutation is a key early driver of tumor growth”
Low-grade glioma is known to have the highest incidence between ages 35 and 42. Because it develops during a period when people are at the center of their working and family lives, the burden is shared not only by the patient but also by families and society. In particular, up to 74% of patients experience seizures, and seizures are reported to persist in more than half (56%) even after surgery. In other words, it is not simply the size of the tumor that is problematic; it is a disease that affects nearly every aspect of life, including daily activities, driving, maintaining employment, and returning to society.
Prof. Jang emphasized the disease burden experienced by patients with low-grade glioma and the need for early targeted therapy.
Prof. Jang said, “Low-grade glioma is difficult to cure and can ultimately recur or progress to high-grade glioma, so patients experience substantial psychological anxiety about worsening disease and recurrence at an important time in their lives.” He added, “Surgical resection, the standard treatment, is often unable to completely remove the tumor, and postoperative radiation therapy and chemotherapy have had limitations because adverse effects such as cognitive impairment can affect quality of life and return to society.”
A target that has recently become a major focus in low-grade glioma treatment is the “IDH mutation.” IDH is an enzyme involved in cellular metabolism; when this gene mutates, an abnormal metabolite called 2-HG, or 2-hydroxyglutarate, is produced. This substance is known to promote tumor growth and malignant transformation.
Prof. Jang said, “IDH mutations are found in more than 80% of patients with grade 2 glioma,” adding that “the World Health Organization (WHO) has also defined IDH mutation as a key diagnostic criterion in its latest revision of the brain tumor classification.” He emphasized, “IDH-mutant gliomas have been reported to increase in tumor size by about 10% every six months, and when tumor size increases by 10%, the risk of death rises by 14%,” and added, “To slow cancer progression, it is important to establish a treatment strategy that suppresses IDH mutations from an early stage.”
Reduced risk of disease progression and death by 65%
Prof. Kim introduced Voranigo’s mechanism of action and the results of a global phase 3 clinical trial. Voranigo is a therapy that simultaneously inhibits IDH1 and IDH2 mutations. It lowers 2-HG levels produced by IDH mutations by more than 90% and was developed to cross the blood–brain barrier and reach brain tumor tissue directly. The blood–brain barrier is a structure that acts as a protective shield preventing external substances from easily entering the brain, and it is considered a major hurdle in developing brain tumor therapies.
In INDIGO, Voranigo’s global phase 3 clinical trial, Voranigo significantly improved progression-free survival compared with placebo. Progression-free survival refers to the length of time during which the cancer does not worsen. At the 6-month follow-up, Voranigo reduced the risk of disease progression or death by 65% versus placebo. It was also shown to delay the time to the next intervention—meaning the point at which additional surgery or radiation/chemotherapy becomes necessary. The risk of needing an intervention before the next treatment decreased by 75%. Even two years after starting treatment, more than 80% of patients remained stable without additional therapy.
Prof. Kim said, “Voranigo is the first IDH-mutant targeted therapy for glioma that dual-inhibits IDH1/2 mutations and crosses the blood–brain barrier to reach the tumor,” adding, “It is highly meaningful as an innovative new drug developed in the brain tumor field for the first time in about 20 years, beyond glioma alone.”
He particularly highlighted the delay in time to the next intervention. Prof. Kim explained, “Being able to postpone the timing of highly toxic chemotherapy and radiation therapy means patients have effectively secured more time to continue normal social and working lives.”
Voranigo also showed clinical benefits in suppressing tumor growth and reducing seizures. In an analysis of tumor growth rate, tumor volume decreased by 1.3% in the Voranigo treatment group, whereas it increased by 14.4% in the placebo group. Prof. Kim said, “Tumor growth rate is an indicator that allows a more precise assessment of disease progression patterns and treatment response,” adding, “A clear tumor growth-suppressing effect was confirmed in the Voranigo treatment group.”
In addition, Voranigo reduced the seizure incidence rate by 64%, and the potential to preserve neurocognitive function was also confirmed. Prof. Kim said, “Based on this clinical value, the National Comprehensive Cancer Network (NCCN) guidelines recommend Voranigo as a Category 1 and preferred regimen,” adding, “With this domestic launch, Voranigo will become a new standard of care for low-grade glioma treatment in South Korea.”
