Study summary

A once-weekly pill shrank tumors in half of children with relapsed BRAF-altered glioma

Tovorafenib approval (FIREFLY-1) · 76 people, phase 2 · FDA accelerated approval April 2024; published April 2025, Clinical Cancer Research

51%response rate

What was tested

Tovorafenib, a RAF inhibitor taken once a week, for children with relapsed or refractory low-grade glioma carrying a BRAF fusion or mutation.

What they found

Tumors shrank in 51%, with responses lasting a median of 13.8 months.
The first FDA-approved drug for pediatric low-grade glioma with BRAF fusions.

What it means

Led to FIREFLY-2, which tests tovorafenib against chemotherapy as first treatment.

Clin Cancer Res, via PubMed ↗ ← Back to the outlook

Last reviewed 2026-11-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.