Servier Plans to Submit NDA for Vorasidenib, a Potential First-in-Class IDH1/2 Inhibitor, Following Phase 3 Data Showing More Than Two-Fold PFS Improvement

▎Edited by the WuXi AppTec content team

Servier showcased the latest clinical data of the potential "first-in-class" IDH1/2 inhibitor vorasidenib for the treatment of IDH-mutant diffuse glioma at the 28th Annual Meeting of the Society for Neuro-Oncology (SNO).The assessment by the blinded independent radiology committee showed that vorasidenib significantly reduced tumor growth. It not only maintained the patients' quality of life but also stabilized neurocognitive function and controlled epilepsy.Based on these results, the company plansSubmit a New Drug Application for vorasidenib in treating IDH-mutant diffuse glioma to the U.S. FDA by the end of 2023.

In the pivotal Phase 3 clinical trial INDIGO, vorasidenib demonstrated a significant improvement in extending progression-free survival (PFS).The median PFS in the vorasidenib treatment group was 27.7 months, compared to 11.1 months in the placebo group.This trial includes patients with residual or recurrent grade 2 gliomas harboring IDH1/2 mutations. The latest analysis shows that vorasidenib not only slowed the tumor growth rate but also led to a reduction in tumor volume, while the tumor volume in the placebo group continued to increase. In the vorasidenib group, the average tumor volume decreased every six months.2.5%, while the average tumor volume of patients in the placebo group increased every six months.13.9%。

Vorasidenib is an orally available, brain-penetrant, and selective investigational dual inhibitor that targets mutant IDH1/2 proteins, positioning it as a potential “first-in-class” drug. It has been granted Breakthrough Therapy Designation by the U.S. FDA.