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罕见病创新药在北京投入临床使用
Cai Jing Wang·2025-07-08 06:55

Core Viewpoint - Fosun Pharma's self-developed drug, Luwo Meitini tablets, has been officially launched for clinical use, marking it as China's first and only targeted drug approved for both adult Langerhans cell histiocytosis (LCH) and neurofibromatosis type 1 (NF1) in children aged 2 and above, providing new treatment options for rare disease patients [1][2]. Group 1: Drug Approval and Clinical Use - Luwo Meitini tablets received approval from the National Medical Products Administration (NMPA) on May 29 and were put into clinical use in Beijing and other cities within a month, addressing the dual challenges of "no available drugs" and "difficult access to drugs" for rare disease patients [1]. - The drug is expected to significantly improve drug accessibility for patients suffering from rare diseases, filling a treatment gap in the domestic market [1]. Group 2: Impact on NF1 Patients - NF1 is a rare genetic disorder that can severely impact patients' quality of life, with surgical interventions often limited in effectiveness [2]. - Luwo Meitini tablets are the first targeted drug approved for NF1 in China, showing promising clinical data with good efficacy and controllable safety, offering new hope for patients who cannot undergo surgery or have unsatisfactory surgical outcomes [2][3]. Group 3: Clinical Data and Efficacy - Luwo Meitini is a novel small molecule targeted drug that inhibits MEK1/2 protein activity, blocking the abnormal activation of the MAPK signaling pathway, thus suppressing tumor cell proliferation and inducing apoptosis [3]. - Phase II clinical trials showed a median follow-up of 15.1 months, with an objective response rate (ORR) of 60.5% and a median time to response (TTR) of 4.7 months, indicating rapid efficacy and good tolerability [3]. Group 4: Broader Implications for Rare Tumors - The drug also addresses a significant unmet need in the treatment of adult LCH and other histiocytic tumors, with an ORR of 82.8% in a Phase II trial for 29 patients, and a median time to response of only 2.9 months [4]. - Future clinical trials are planned for additional conditions, including low-grade gliomas and pediatric LCH, indicating the potential for broader applications of Luwo Meitini [4].