Vorasidenib: Expanding Applications in Glioma Treatment

Creator: Dr. Saad Mohammad
Published: 2025-11-24T19:23:07-05:00

: Dr. Krishna Suthar Medical Source: Dr. Steve Braunstein
UCSF Health Written by: Dr. Saad Mohammad
NYU Langone

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Vorasidenib’s Role in Glioma Care Expands, but Key Questions on Timing and Benefit Remain

A once daily pill called Vorasidenib, originally approved for recurrent low-grade IDH-mutant gliomas, is now being explored across much broader treatment settings.
Clinicians are increasingly considering it before surgery, after surgery, upfront, or at recurrence, though evidence for optimal timing remains incomplete.
Experts urge cautious, individualized use as ongoing research aims to clarify who benefits most and how best to sequence the therapy.

The introduction of vorasidenib, an oral targeted therapy for IDH-mutant gliomas, has reshaped how clinicians think about treating low-grade and select recurrent brain tumors. While its initial approval came from the INDIGO trial, published in The New England Journal of Medicine, real-world use of this drug is rapidly expanding.

“So the original indication and approval for this drug was based upon the INDIGO trial, and that was specifically for patients who had recurrent progressive lower-grade gliomas following surgery,” Dr. Steve Braunstein, an oncologist from the University of California at San Francisco, tells SurvivorNet.

“We’re fortunate enough that that indication expanded significantly in prior months to be able to include patients with IDH-mutant tumors… in various settings.”

This shift reflects the growing belief that vorasidenib may play a broader role in glioma care than initially understood.

Understanding INDIGO Trials

Expanding Clinical Use

What began as a treatment for post-surgical recurrent low-grade glioma is now being explored across the entire disease spectrum.

Dr. Braunstein addresses these new uses when he says, “This is now something that could be used in a neoadjuvant setting, potentially before surgery.

“It could be used following surgeries, it could be used or considered in the upfront setting. It could be considered in the recurrent setting even after radiation.”

These expanded applications are driven by interest in using vorasidenib earlier—or more strategically—during treatment.

Unanswered Questions About Timing and Sequencing

Despite enthusiasm, major questions remain. Evidence for some uses is emerging, but far from complete which Dr. Braunstein is careful to draw caution
to.

Dr. Braunstein says, “We don’t have data, although it’s emerging… to really understand what is the optimal timing and sequencing of these agents for patients who have IDH-mutant gliomas.”

Key uncertainties include:

When is the best time to start treatment?
Does neoadjuvant (pre-surgical) use impact outcomes?
How well does the drug work after radiation or chemotherapy?
Will long-term use change tumor behavior?
What if any, long-term cognitive and quality of life impacts are there with the use of Vorasidenib?

Personalized Medicine and the Tremendous Value of Molecular Testing Gliomas

Real-World Caution

Clinical judgment remains critical, even with promising results. Dr. Braunstein is careful to discuss that treatment remains an individualized decision, explaining, “When we apply this to the patient care scenarios, we try to be very deliberate… about the current evidence and where there may be uncertainty.

“Despite the strong results of the INDIGO trial, we recognize that not every patient with IDH mutation may benefit. Patients who’ve been through other forms of therapy—who didn’t exactly fit the characteristics of those included on trial—may not yield that benefit,” Dr. Braunstein states.

Ongoing Research

“But again, many groups are investing significant time and resources to ask those questions on trial so that we can be more informed about who is best served by these types of agents and what is the optimal timing and sequencing to deliver them,” adds Dr. Braunstein.

What Patients Should Ask Their Doctor

Do I have an IDH1 or IDH2 mutation confirming eligibility for vorasidenib?
Is vorasidenib appropriate for me now—before surgery, after surgery, or only
if the tumor progresses?
How closely does my case match the patients enrolled in the INDIGO trial?
What side effects should I expect, and how will we monitor them?
If I’ve had radiation or chemotherapy, will vorasidenib still be effective?
Could this treatment delay or replace radiation or chemotherapy?
What are the uncertainties about long-term outcomes?
Are clinical trials available that might better match my situation?

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Dr. Mohammad is an anesthesiologist at NYU Langone. Read More