复星自研罕见病创新药发布最新研究成果 已在多省市投入临床使用

Group 1 - The core viewpoint of the articles highlights the significant research findings of the MEK1/2 inhibitor FCN-159 (Luvoxetine) developed by Fosun Pharma for treating relapsed/refractory Langerhans cell histiocytosis (LCH) in children, which were presented at the 30th European Hematology Association (EHA) annual meeting in 2025 [1][2] - FCN-159 has completed its first batch of shipments and is now officially in clinical use across multiple hospitals in provinces such as Shanghai, Beijing, Guangdong, Shandong, Jiangsu, and Hunan [1] - FCN-159 is a highly selective oral small molecule inhibitor that blocks the abnormal activation of the MAPK signaling pathway, inhibiting tumor cell proliferation and inducing apoptosis [1] Group 2 - The II phase study results of FCN-159 for treating pediatric LCH were presented in poster form at the EHA conference, demonstrating its efficacy and safety [2] - This multicenter, open-label, single-arm II phase study, led by Professor Zhang Rui from Beijing Children's Hospital, aimed to evaluate the effectiveness and safety of FCN-159 in treating relapsed/refractory LCH in children [2] - Preliminary results indicate that FCN-159 shows significant efficacy and good tolerability in treating refractory/relapsed LCH in children, with no reports of treatment-related adverse events (TRAEs) leading to discontinuation [2]