Bei Ke Cai Jing·2025-06-19 09:01Core Viewpoint - The launch of Luwo Meitini tablets marks a significant advancement in the treatment of rare diseases in China, specifically targeting LCH and NF1, providing new therapeutic options for patients [1][2][3]. Group 1: Product Overview - Luwo Meitini tablets are the first and only targeted drug approved in China for both adult LCH and NF1 in children aged 2 and above [1]. - The drug is an innovative small molecule developed by Fosun Pharma, selectively inhibiting MEK1/2 protein activity to block the abnormal activation of the MAPK signaling pathway, thereby suppressing tumor cell proliferation and inducing apoptosis [2]. Group 2: Clinical Data and Efficacy - Clinical trial data shows a median follow-up time of 15.1 months, with a best objective response rate (ORR) of 60.5% and a median time to response (TTR) of 4.7 months, indicating effective and controllable safety [2]. - NF1 is a rare autosomal dominant genetic disorder, with a significant disease burden that can lead to pain, mobility issues, and disfigurement, necessitating the development of targeted therapies [2]. Group 3: Future Prospects - In addition to the current indications, Luwo Meitini is undergoing clinical trials for low-grade gliomas, extracranial arteriovenous malformations, and pediatric LCH, with the potential to expand its application to more disease areas [4].