The FDA approval of dabrafenib plus trametinib in March 2023 provided a much-needed targeted therapy option for patients with BRAF^V600E-mutant low-grade glioma (LGG). Nonetheless, as a type I kinase inhibitor, dabrafenib is ineffective in patients with BRAF fusions, including KIAA1549–BRAF, which has been detected in ~35% of paediatric patients with LGG. Now, data from the phase II FIREFLY-1 trial demonstrate the efficacy of the type II RAF inhibitor tovorafenib in paediatric patients (1–24 years of age) with treatment-refractory LGG harbouring BRAF fusions or BRAF^V600E mutations.

中文翻译：

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托沃拉非尼对含有 BRAF 融合的低级别胶质瘤有效

FDA 于 2023 年 3 月批准达拉非尼联合曲美替尼，为BRAF ^V600E突变低级别胶质瘤 (LGG)患者提供了急需的靶向治疗选择。尽管如此，作为 I 型激酶抑制剂，达拉非尼对BRAF融合患者无效，包括KIAA1549 – BRAF，已在约 35% 的 LGG 儿科患者中检测到。现在，II 期 FIREFLY-1 试验的数据证明了 II 型 RAF 抑制剂托沃拉非尼 (tovorafenib) 对携带BRAF融合或BRAF ^V600E突变的难治性 LGG 儿科患者（1-24 岁）的疗效。