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news.The US Food and Drug Administration (FDA) has accepted Servier’s New Drug Application (NDA) filing and priority review for vorasidenib, a new treatment for isocitrate dehydrogenase (IDH)-mutant diffuse glioma.
The regulatory milestones are based on data from the Phase III INDIGO clinical trial of vorasidenib in glioma patients. Credit: Milad Fakurian on Unsplash.
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Concurrently, the European Medicines Agency (EMA) granted accelerated assessment for the marketing authorization application (MAA) of the same drug.
On receipt of approval, vorasidenib would become the sixth therapy to receive approval for IDH-mutant cancers.
A decision on the approval from the FDA under the Prescription Drug User Fee Act (PDUFA) is anticipated by 20 August 2024 while approval by the European Commission is expected in the second half of this year.
These regulatory milestones are based on data from the global, randomised, double-blind placebo-controlled Phase III INDIGO clinical trial of vorasidenib.
It enrolled subjects with residual or recurrent grade two glioma carrying an IDH1/2 mutation, who had previously undergone surgery.
Data showed that the targeted therapy demonstrated significant improvements in progression-free survival (PFS) and time to next intervention (TTNI), meeting primary and key secondary endpoint, respectively.
Median PFS was 27.7 months and 11.1 months for vorasidenib and placebo arms, respectively.
Additionally, vorasidenib demonstrated a reduction in tumour volume, with a mean decrease of 2.5% every six months, contrasting with a mean increase of 13.9% in the placebo arm.
The safety profile of vorasidenib was in line with prior Phase I study data, indicating that the drug was well-tolerated.
Servier Cancer Metabolism Global Development Oncology & Immuno-Oncology head Susan Pandya said: “In the realm of glioma treatment, innovation has been stagnant for nearly a quarter-century, posing challenges for patients who, post-surgery, may opt to defer treatment due to concerns around potential toxic side effects. As a drug specifically designed to be highly blood-brain barrier penetrant, vorasidenib has demonstrated clinically meaningful efficacy in patients with IDH1/2 mutant gliomas alongside a consistently manageable safety profile.
“This promising outcome brings hope to patients grappling with IDH-mutant diffuse gliomas, offering a potential breakthrough for those eagerly awaiting a new therapeutic option.”