FDA Approves First Targeted Therapy for IDH-Mutant Glioma with Companion Diagnostic

The U.S. Food and Drug Administration (FDA) has approved Thermo Fisher’s Oncomine Dx Target Test, a next-generation sequencing (NGS)-based companion diagnostic, to identify patients eligible for Servier's VORANIGO® (vorasidenib). This is the first targeted therapy for Grade 2 IDH-mutant glioma, offering a new treatment option for patients with intractable brain tumors.

The test detects key mutations in IDH1 and IDH2, enabling personalized treatment, and is available in 19 countries, enhancing precision medicine globally.

"VORANIGO is the first and only targeted therapy for patients living with grade 2 IDH mutant glioma, a relentless and incurable type of brain cancer," said David K. Lee, CEO of Servier Pharmaceuticals. “Identifying key driver mutations is essential to help the right patients find the right treatment, at the right time.”

Targeted Treatment for a Difficult Condition

Gliomas are the most common malignant primary brain tumors, accounting for approximately 81% of cases. About 20% of gliomas harbor mutations in IDH genes. Until now, treatment options for Grade 2 IDH-mutant gliomas have been limited, with little progress in nearly 25 years. 

VORANIGO, an IDH1 and IDH2 inhibitor, changes that by offering a targeted approach for both adult and pediatric patients aged 12 years and older, following surgical interventions such as biopsy or resection.

This new therapy, paired with the Oncomine Dx Target Test, offers hope for glioma patients who previously had no targeted treatment options. VORANIGO’s approval could significantly improve outcomes for those with IDH-mutant gliomas.

Kathy Davy, president of clinical next-generation sequencing at Thermo Fisher Scientific, emphasized: “As the healthcare system works to realize the impact of precision medicine, patients must have access to the proper testing that helps unlock targeted treatment options based on their unique genomic profiles. This access is the driving motivation behind the extensive work we do with pharma partners to help connect the right patients to new therapies as soon as they are approved.”

A Milestone in Precision Medicine

The Oncomine Dx Target Test helps doctors identify patients who may benefit from VORANIGO by detecting IDH1 and IDH2 mutations. This test has already been approved for multiple cancers, including non-small cell lung cancer, cholangiocarcinoma, and thyroid cancer. It enables simultaneous biomarker testing, quickly matching patients with the most appropriate therapies based on their genetic profile.

Approved and reimbursed by insurers in 19 countries, including the U.S., Japan, and South Korea, the Oncomine Dx Target Test covers more than 550 million people globally. The FDA approval of VORANIGO will further expand its use, allowing patients to receive faster and more precise treatments for IDH-mutant gliomas.