Servier Pharmaceuticals’ vorasidenib on Tuesday secured the FDA’s green light for the treatment of patients with grade 2 gliomas carrying mutations in the IDH gene.
The FDA on Tuesday signed off on Servier Pharmaceuticals’ oral treatment vorasidenib for the treatment of patients with grade 2 gliomas carrying mutations in the IDH gene. The drug will be marketed under the brand name Voranigo.
Tuesday’s regulatory victory makes Voranigo the first targeted and systemic therapy for patients with grade 2 astrocytoma or oligodendroglioma with susceptible IDH1 or IDH2 mutations, according to the FDA’s announcement. Voranigo is indicated for patients aged 12 years and older who have undergone surgical procedures, such as biopsy, subtotal resection or gross total resection.
Servier Chief Commercial Officer Arjun Prasad in a statement called Voranigo’s approval “an enormous leap forward in cancer care, and a defining moment for people living with grade 2 IDH-mutant glioma.” The drug is the “first breakthrough” in this indication in almost 25 years, Prasad said. Servier has yet to announce a launch price for Voranigo.
Designed for once-daily oral dosing, Voranigo is a brain-penetrant small molecule inhibitor of the IDH1 and IDH2 enzyme, according to its label. Through this mechanism of action, the drug prevents the build-up of the cancer-causing metabolite D-2-hydroxyglutarate. Voranigo was originally discovered and developed by Agios Pharmaceuticals, which Servier bought in December 2020 for $2 billion plus royalties.
Tuesday’s approval triggers a $200 million milestone payment from Servier to Agios, according to its second-quarter 2024 business report. The Massachusetts biotech will also receive a $905 million payment from Royalty Pharma which recently purchased its royalty rights to Voranigo. Agios retains its 3% royalty entitlement on annual net sales of Voranigo that exceed $1 billion.
Voranigo’s approval was backed by data from the Phase III INDIGO study, a randomized, double-blinded and placebo-controlled trial. The drug elicited a clinically meaningful improvement in progression-free survival versus placebo, cutting the risk of progression or death by 61%. This effect was highly statistically significant, with a p-value less than 0.001, according to Servier.
Voranigo also elicited significant benefits versus placebo in terms of time to next intervention and tumor volume. The late-stage study also found it to be safe overall, with a tolerability profile that mirrored Phase I findings.
Beyond glioma, Servier is also developing vorasidenib—Voranigo’s active ingredient—for other solid tumors, either alone or in combination with Merck’s Keytruda (pembrolizumab).
