Tovorafenib

Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors.

Tovorafenib is being investigated as a front-line therapy for patients newly diagnosed with progressive pediatric low-grade glioma (pLGG) in the pivotal Phase 3 FIREFLY-2/LOGGIC randomized clinical trial. Tovorafenib is also being evaluated as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1).

Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission for the treatment of glioma.

OJEMDA^TM (tovorafenib) has been approved by the FDA for children with BRAF-altered, relapsed or progressive pLGG.