又一罕见病药物在华获批，用于全身型重症肌无力

Core Insights - The approval of Zilucoplan (brand name Zhuobeike) by the National Medical Products Administration marks a significant advancement in the treatment of generalized myasthenia gravis (gMG) in adults, being the first and only subcutaneous self-administered C5 complement inhibitor with dual inhibition mechanisms [1][3] Industry Overview - Generalized myasthenia gravis is a rare, chronic autoimmune disease affecting approximately 1.2 million patients globally, with around 220,000 cases in China. Symptoms include diplopia, ptosis, dysphagia, and life-threatening respiratory muscle weakness [2] - The disease is characterized by fluctuating symptoms and challenges in long-term management due to limitations in existing treatments, which often have significant side effects and unstable efficacy [2] Product Impact - Zilucoplan offers a convenient administration method, allowing patients to self-inject in just 5-8 seconds, thus reducing the burden of hospital visits and enhancing patient independence in disease management [3] - Clinical studies indicate that Zilucoplan can rapidly improve symptoms within a week and maintain stable efficacy and quality of life over long-term treatment [3]