FDA Approves Vorasidenib as First Targeted Therapy for G2 IDH-Mutant Glioma

The FDA has approved Servier's VORANIGO® (vorasidenib) for treating Grade 2 astrocytoma or oligodendroglioma in patients 12 and older with IDH mutations. This first targeted therapy demonstrated significant progression-free survival benefits in the INDIGO trial.

The U.S. Food and Drug Administration (FDA) has approved Servier's VORANIGO® (vorasidenib), marking a significant advancement in the treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation in patients aged 12 and older. The approval followed impressive results from the INDIGO Phase 3 trial, showing a median progression-free survival of 27.7 months, significantly outperforming the placebo's 11.1 months.

Developed by Servier Pharmaceuticals, vorasidenib is the only FDA-approved targeted therapy specifically for Grade 2 IDH-mutant glioma, a type of brain cancer that can severely impact neurological functions and quality of life. It offers a convenient once-daily pill to manage these types of brain tumors.

“Today’s approval of VORANIGO is an enormous leap forward in cancer care, and a defining moment for people living with Grade 2 IDH-mutant glioma. It is the first breakthrough in this specific disease area in nearly 25 years, offers patients unprecedented improvement in progression free survival. We are proud to deliver this first-of-its-kind therapy to patients in need, and we remain committed to bringing innovative targeted therapies to people with cancer,” said Arjun H. Prasad, Chief Commercial Officer, Servier Pharmaceuticals.

A New Era in Cancer Therapy

This approval follows the impressive results from the INDIGO Phase 3 clinical trial, which demonstrated significant improvements in progression-free survival, with a well-tolerated safety profile. The trial revealed that patients treated with vorasidenib  experienced a median progression-free survival of 27.7 months, significantly longer than the 11.1 months observed in patients receiving a placebo. These findings, published in The New England Journal of Medicine, highlight the potential of vorasidenib to change the treatment landscape for glioma patients.

The Science Behind Vorasidenib

Vorasidenib functions by inhibiting mutant enzymes IDH1 and IDH2, which are often found in glioma cells. In healthy cells, these enzymes are involved in the cellular metabolism and energy production. However, mutations in these enzymes can prevent cells from differentiating properly, leading to uncontrolled growth and cancer development. By targeting these mutant enzymes, VORANIGO® helps to manage the growth of the tumor effectively.

Patient-Centric Drug Development

Vorasidenib is available in a convenient once-daily pill form, simplifying treatment for patients and potentially improving their quality of life. This is especially significant considering the younger demographic often affected by these tumors, typically diagnosed in adults under 50 years of age.  

Collaborative International Review

The review of VORANIGO® was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence, which facilitates concurrent submission and review of oncology drugs among international partners. This collaborative effort included regulatory bodies from Australia, Brazil, Canada, Switzerland, and Israel, emphasizing the global importance and potential impact of this new therapy. 

With this approval, Servier Pharmaceuticals solidifies its position as a leader in the development of targeted therapies for IDH-mutant cancers, with VORANIGO® being their sixth approval in this category.