FDA Approves Servier’s Voranigo for Brain Cancer Treatment in Adults and Children

The US Food and Drug Administration (FDA) has granted approval for Servier Pharmaceuticals' IDH1/2 dual inhibitor, Voranigo (vorasidenib), to treat a prevalent form of brain cancer in adults and pediatric patients aged 12 and older. Voranigo, administered as a once-daily tablet, is intended for patients with grade 2 astrocytoma or oligodendroglioma possessing a susceptible IDH1 or IDH2 mutation post-surgery.

Diffuse gliomas with IDH mutations are the most frequent malignant primary brain tumors diagnosed in adults under 50 years of age. Current therapies are unable to cure these tumors, which continue to grow and infiltrate normal brain tissue if left untreated. Voranigo is now the first FDA-approved targeted treatment for grade 2 IDH-mutant glioma. It helps manage the disease by inhibiting the activity of the mutant IDH1 and IDH2 enzymes associated with various cancers.

The FDA’s decision was based on positive results from the late-stage INDIGO clinical trial. The trial demonstrated that Voranigo significantly extended progression-free survival (PFS) and time to next intervention (TTNI) compared to a placebo. Specifically, the median PFS for patients treated with Voranigo was 27.7 months, compared to 11.1 months for those in the placebo group. Additionally, TTNI was not reached for Voranigo but was 17.8 months for the placebo group.

Further, Voranigo was shown to reduce tumor volume by an average of 2.5% every six months, while tumor volume increased by an average of 13.9% in the same period for patients in the placebo arm. These findings highlight the drug’s potential in effectively managing and slowing the progression of IDH-mutant gliomas.

Arjun Prasad, chief commercial officer at Servier, emphasized the significance of this approval, describing it as "an enormous leap forward in cancer care." Prasad noted that Voranigo is the first major breakthrough in this specific disease area in nearly 25 years, offering patients remarkable improvements in progression-free survival.

Echoing Prasad’s sentiments, Ralph DeVitto, president and chief executive officer of the American Brain Tumor Association, remarked that patients with grade 2 IDH-mutant gliomas have long faced the grim reality of an incurable disease with very limited treatment options post-surgery. According to DeVitto, the FDA approval of Voranigo represents a monumental breakthrough in glioma treatment, bringing renewed hope to patients and their families battling this relentless disease.

In summary, the FDA’s approval of Voranigo signifies a major advancement in the treatment of grade 2 IDH-mutant gliomas. By targeting and inhibiting the mutant IDH1 and IDH2 enzymes, Voranigo offers a new, effective option for managing this challenging form of brain cancer, providing significant improvements in both progression-free survival and overall quality of life for patients.