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Tovorafenib is an oral, brain-penetrant, highly-selective type II RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. It inhibits mutant BRAF serine-threonine kinase (including V600E and V600D mutations), wild-type BRAF, and wild-type CRAF kinases. Tovorafenib blocks dysregulated signaling pathways related to cancer cell growth and survival without inducing paradoxical activation of the MAPK pathway. It is primarily indicated for pediatric patients (6 months and older) with relapsed or refractory low-grade glioma harboring a BRAF fusion/rearrangement or BRAF V600 mutation[1][2][3][4][5][7][9]. The drug was granted accelerated approval by the FDA in April 2024 as the first systemic therapy for this indication[7]. Tovorafenib has also received Breakthrough Therapy and Orphan Drug designations from regulatory agencies[4].
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