FDA Approves Servier’s VORANIGO for IDH-Mutant Grade 2 Glioma

The US Food and Drug Administration (FDA) has granted approval for Servier’s VORANIGO for treating patients aged 12 years and older who have Grade 2 astrocytoma or oligodendroglioma with mutations in the isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) genes. This medication is indicated for patients who have undergone surgical procedures, including biopsy or gross total resection.

VORANIGO is a once-daily oral medication that functions as an inhibitor for both IDH1 and IDH2 enzymes. The FDA’s decision is based on data from the global, randomized, double-blind, placebo-controlled Phase III INDIGO clinical trial. The trial involved patients who had residual or recurrent Grade 2 glioma with an IDH1/2 mutation and had only received surgical treatment.

The primary outcome of the trial was progression-free survival (PFS), assessed by a blinded independent review committee. The secondary endpoint was the time to the next intervention, evaluated during a predetermined second interim analysis. The results indicated that VORANIGO enhanced PFS and postponed the need for further interventions compared to the placebo. The safety profile of VORANIGO was consistent with what had been seen in Phase I trials, and it was generally well tolerated. The most common adverse reactions reported were fatigue, Covid-19, musculoskeletal pain, diarrhea, and seizures.

Arjun Prasad, the Chief Commercial Officer of Servier Pharmaceuticals, commented on the approval, stating, “The approval of VORANIGO marks a substantial advancement in cancer treatment and a significant milestone for those living with Grade 2 IDH-mutant glioma. This breakthrough therapy, the first in nearly 25 years for this specific disease, offers patients significant improvement in progression-free survival. We are honored to provide this novel therapy to those in need and remain committed to delivering innovative targeted treatments to cancer patients.”

In October 2023, the FDA also approved Tibsovo (ivosidenib tablets) by Servier for treating IDH1-mutated relapsed or refractory myelodysplastic syndromes.