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The FDA has granted breakthrough therapy designation to plixorafenib, an oral targeted drug, for patients with recurrent high-grade gliomas with a BRAF V600E mutation. This designation allows closer collaboration with the FDA and is intended to shorten development time.
Early clinical results show encouraging activity, particularly in brain tumors. In a small predefined subgroup of 9 patients with BRAF V600E-mutated primary CNS tumors (including high-grade gliomas) who had not received prior similar targeted therapies, 67% had tumor shrinkage and more than 75% experienced clinical benefit (shrinkage or stable disease).
Across all BRAF V600-mutated solid tumors, the response rate was 42%, with a median response duration of 17.8 months and over 70% experiencing clinical benefit.
The drug has shown a generally favorable safety profile, and an ongoing phase 2 trial is further evaluating its effectiveness in BRAF-mutated cancers, including brain tumors. While these results are promising, larger studies are needed to confirm efficacy and response durability in high-grade glioma.