# Tovorafenib

Source: https://onco.cc/drugs/tovorafenib/  
OnCo record `tovorafenib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Tovorafenib is a pill for the most common childhood brain tumour, low-grade glioma driven by BRAF changes, approved in 2024.

## Summary

FDA accelerated approval 23 April 2024 for relapsed/refractory paediatric low-grade glioma (age ≥6 months) with BRAF fusion/rearrangement or V600 mutation (FIREFLY-1: ORR ~51% by RANO-HGG). European conditional approval April 2026 (Ipsen, regardless of BRAF alteration type). Type II RAF inhibitor active against KIAA1549-BRAF fusions where type I inhibitors cause paradoxical activation. Phase 3 LOGGIPY-2 in first line versus chemotherapy. Day One Biopharmaceuticals.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-06
- Brand: Ojemda
- Modality: Small-molecule type II RAF inhibitor
- Mechanism: Pan-RAF type II inhibitor binding the DFG-out conformation; blocks monomeric and dimeric BRAF signalling.
- Approvals: US 2024: Relapsed/refractory BRAF-altered paediatric low-grade glioma (accelerated); EU 2026: Relapsed/refractory paediatric low-grade glioma (conditional)

## Sources

- FDA approval: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-tovorafenib-patients-relapsed-or-refractory-braf-altered-pediatric

## Connected records

- biomarkers: [BRAF class II and class III mutations (non-V600)](https://onco.cc/biomarkers/braf-class-ii-iii/), [BRAF fusion or rearrangement](https://onco.cc/biomarkers/braf-fusion/)
- cancers: [Brain and spinal cord tumours (all types)](https://onco.cc/cancers/brain-tumours/), [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [Glioma & glioblastoma](https://onco.cc/cancers/glioblastoma/), [Optic pathway glioma](https://onco.cc/cancers/optic-pathway-glioma/), [Paediatric low-grade glioma](https://onco.cc/cancers/paediatric-low-grade-glioma/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [BRAF](https://onco.cc/targets/braf/)
- companies: [Day One Biopharmaceuticals](https://onco.cc/companies/day-one-biopharmaceuticals/), [Ipsen](https://onco.cc/companies/ipsen/)
- pathways: [Glioma (KEGG map)](https://onco.cc/pathways/glioma-signalling/), [RAS / RAF / MEK / ERK (MAPK)](https://onco.cc/pathways/ras-mapk/)
- trials: [DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)](https://onco.cc/trials/nct05566795/), [FIREFLY-1](https://onco.cc/trials/firefly-1/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [The brain: barrier and sanctuary](https://onco.cc/bottlenecks/b-brain-delivery/)
- ideas: [Develop drugs in children first when the target is a children's target](https://onco.cc/ideas/idea-bio2-paediatric-first-development/)
- terms: [RACE for Children Act](https://onco.cc/terms/race-for-children-act/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

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