Voranigo is Safe and Tolerable in High-Grade IDH-Mutant Gliomas

Voranigo (vorasidenib), a drug for certain brain tumors, was safe and well toleratedin patients with grade 3 or 4 IDH-mutant gliomas, according to short-term data presented at the 2025 Society for Neuro-Oncology (SNO) Annual Meeting in Honolulu, Hawaii.

Based on findings from the nine-month review of 32 patients, 53.1% of patients had stable disease while taking Voranigo, and patients stayed on the medication for an average of 3.5 months. Additionally, at the time of follow-up, more than half of the patients were still receiving Voranigo, with 19 individuals continuing treatment.

“Overall, we saw that [Voranigo] was well tolerated and provided disease stability in approximately half of our patients over a median treatment duration of 3.5 months,” said study presenter Amanda Knott, noting that these initial outcomes show early signals of tolerability for those with IDH-mutant high-grade gliomas.

Knott is the assistant program director of Investigational Drug Services, where she is also an instructor in pharmacy, as well as a research pharmacist at Mayo Clinic College of Medicine & Science in Rochester, Minnesota.

Patient Demographics, Prior Treatments, and Treatment Duration With Vorasidenib in Grade 3 and 4 IDH-Mutant Gliomas

The study included 32 patients with a median age of 39.5 years. There were 17 men and 15 women. Tumor types included grade 3 astrocytoma (13 patients), grade 3 oligodendroglioma (11 patients) and grade 4 astrocytoma (8 patients). Most tumors showed “contrast enhancement” on imaging, which means the tumors were more visible with certain scans, while two tumors did not.

Most individuals were heavily pretreated. Surgical resection was the most common prior modality (30 patients), followed by radiation (28 patients) and cytotoxic chemotherapy (25 patients). Additional systemic therapies included Tibsovo (ivosidenib) in eight patients, Avastin (bevacizumab) in five patients and Keytruda (pembrolizumab) in three patients. Four patients began Voranigo with no history of prior systemic therapy.

Patients stayed on Voranigo for a median of 3.5 months (101.5 days), ranging from 18 days to 255 days. Nearly 60% of patients were still on the medication at their last follow-up, which suggests that for some, Voranigo may help control tumor growth or maintain stability.

Safety Profile and Side Effects With Voranigo in Grade 3 and Grade 4 IDH-Mutant Gliomas

Voranigo was generally safe and aligned with expected toxicities. Laboratory abnormalities were predominantly low grade. Transaminitis (having high levels of liver enzymes) occurred in 12 patients, all grade 1 (mild), and no high-grade liver enzyme elevations were observed. Lymphopenia developed in two individuals, both of whom previously received multiple alkylating agents. No additional hematologic toxicities were identified.

Other side effects included fatigue, which was seen in nine patients, headache in four patients, decreased appetite in two patients, nausea in two patients and myalgias in one patient. One case of myalgias (muscle pain) led to treatment discontinuation, representing the only therapy-limiting toxicity reported in the cohort.

Knott described the overall side effect profile as “as expected,” reiterating that Voranigo was “well tolerated.”

However, progression occurred in 34.4% of patients. Notably, two patients have not yet reached their first disease assessment and two patients died during the observation period.

Ongoing Studies Will Clarify Durability, Long-Term Safety and Clinical Integration

Knott concluded the presentation by stating that: “Our conclusions are somewhat limited based on the short term follow up, so longer-term follow up is needed, and prospective studies are ongoing for [Voranigo].”

She emphasized the need for extended observation to better characterize durability of response and long-term tolerability of Voranigo for both grade 3 and grade 4 IDH-mutant gliomas.

Reference

1. “Experience with vorasidenib in patients with grade 3 or 4 IDH-mutant gliomas,” by Amanda Knott. Presented at 2025 Society of Neuro-Oncology Annual Meeting; Nov. 19-23, 2025; Honolulu, HI. Abstract NCOG-44.

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