Tovorafenib appears to have anticancer activity when used to treat patients with recurrent or progressive pediatric low-grade glioma, according to data from the pivotal phase 2 FIREFLY-1 trial.1
About Tovorafenib
Tovorafenib is an investigational, orally administered, highly selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway.
The FIREFLY-1 clinical trial is evaluating tovorafenib in patients aged 6 months to 25 years with recurrent or progressive low-grade glioma. Patients are required to have relapsed or progressive low-grade glioma with documented known activating BRAF alteration, at least 1 prior line of systemic therapy with evidence of radiographic progression, and at least 1 measurable lesion.
Among 77 patients who received treatment with tovorafenib, 64% responded to treatment. Researchers reported a complete response rate of 4% and a partial response rate of 59%. Additionally, 28% of patients experienced stable disease, and the clinical benefit rate was 91%.
Tovorafenib was well tolerated, and the most common side effects related to the agent included change in hair color, fatigue, and a maculopapular rash.
References
1. Day One announces topline data from pivotal phase 2 FIREFLY-1 trial demonstrating meaningful responses with tovorafenib (DAY101) in recurrent or progressive pediatric low-grade glioma. News release. Day One. January 8, 2023. Accessed January 9, 2023. https://bit.ly/3VR4id7.
2. A study to evaluate DAY101 in pediatric and young adult patients with relapsed or progressive low-grade glioma and advance solid tumors (FIREFLY-1). ClinicalTrials.gov. Updated October 3, 2022. Accessed January 9, 2023. https://clinicaltrials.gov/ct2/show/NCT04775485
