Tovorafenib (DAY101) is an investigational, oral, brain-penetrant, highly-selective type II RAF kinase inhibitor.
Dysregulation of the MAPK pathway has been shown to occur in many cancer types and it is one of the most commonly mutated oncogenic pathways in cancer.
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 may offer an important alternative for people with primary brain tumors or brain metastases of solid tumors.
Tovorafenib has been studied in over 300 patients to date. Currently tovorafenib is under evaluation in a pivotal Phase 2 clinical trial (FIREFLY-1) for the treatment of pediatric low-grade glioma (pLGG). pLGG is the most common form of childhood brain cancer. We have also initiated a Phase 1/2 study (FIRELIGHT-1) with tovorafenib in patients with recurrent or progressive solid tumors with activating RAF alterations.
Tovorafenib has the potential to become an important alternative for people with primary brain tumors or brain metastases of solid tumors.
In October 2023, Day One announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application for tovorafenib monotherapy in relapsed or progressive pLGG for Priority Review. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
Tovorafenib has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. The FDA has also granted Rare Pediatric Disease Designation to tovorafenib for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. Tovorafenib has also received Orphan Drug designation from both the FDA and the European Medicines Agency (EMA) for the treatment of malignant glioma.