又一罕见病药物进入中国 已有同类全身型重症肌无力产品通过今年国谈初审目录

Core Insights - UCB's drug, Zilucoplan (brand name: Zhuobeike), has received approval from China's National Medical Products Administration (NMPA) for the treatment of adult generalized myasthenia gravis (GMG) patients who are positive for acetylcholine receptor (AChR) antibodies [1] - Zilucoplan is the first and only subcutaneous self-administered C5 complement inhibitor, which can effectively block complement-mediated neuromuscular junction damage, allowing patients to self-administer the injection at home in just 5-8 seconds [1] - The prevalence of generalized myasthenia gravis in China is approximately 220,000 patients, with AChR antibodies detected in 80%-90% of cases [1] Drug Market Overview - Several drugs for generalized myasthenia gravis have entered the Chinese market recently, including UCB's other drug, Rozanolixizumab (brand name: Yudige), which was approved in March and covers both AChR-positive and MuSK-positive patients [1] - AstraZeneca's long-acting C5 complement inhibitor, Riluzole (brand name: WeiliRui), was approved in April for AChR-positive adult GMG patients [2] - Rongchang Biotech's Taitasip (brand name: Tai'ai) was also approved in May for AChR-positive adult GMG patients [2] - The number of subcutaneous injection options remains limited compared to intravenous formulations, with the approval of Efgartigimod (brand name: WeiliJia) in July last year, which requires a longer administration time of 30-90 seconds [2]