On October 11, 2025, UCB S.A. (hereinafter referred to as UCB) announced that the NMPA had approved the marketing authorization for Zilucoplan (brand name: ZHUOBEIKE®), which is used in combination with conventional therapeutic drugs for the treatment of adult generalized myasthenia gravis (gMG) patients with positive anti-acetylcholine receptor (AChR) antibodies.

Zilucoplan is the world’s first and only new-generation C5 complement inhibitor that can be self-administered via subcutaneous injection and possesses dual inhibitory effects. It prevents the cleavage of C5 into C5a and C5b, hinders the formation of the terminal complement complex C5b-9, and simultaneously blocks the binding of C5b and C6, effectively stopping the complement cascade reaction. As an innovative drug with a dual inhibitory mechanism, it can effectively block complement-mediated neuromuscular junction damage. Patients can complete subcutaneous injection at home in just 5-8 seconds, which not only reduces the burden of traveling to and from the hospital but also helps them gain greater convenience and independence in disease management.

gMG is a rare, chronic, and heterogeneous autoimmune disease primarily characterized by neuromuscular junction dysfunction. It can occur at any age. Since the 1950s, due to improved diagnostic accuracy and reduced mortality rates, the prevalence of this disease has continued to rise. The existing traditional treatment methods have limitations, including significant drug side effects, unstable therapeutic efficacy, and limited accessibility, making it difficult for patients to manage their condition continuously.

The approval of Zilucoplan was based on the Phase III RAISE study (NCT04115293), which showed that in patients with AChR antibody-positive gMG, the drug significantly improved MG-ADL (Myasthenia Gravis Activities of Daily Living Scale) and QMG scores (Quantitative Scoring System for Myasthenia Gravis), with statistically significant differences compared to placebo, and demonstrated good safety. The trial results indicated that patients could experience significant improvements in breathing, swallowing, and speaking within one week after medication, and long-term treatment could bring stable and lasting efficacy, while reducing dependence on intravenous immunoglobulin (IVIg) and plasma exchange (PE).

The approval status of Zilucoplan globally is presented in Table 1 below, with its global sales in 2024 amounting to USD 77.6 million.

Table 1

UCB has not yet published the Chinese patent information for Zilucoplan on the China Drug Patent Information Registration Platform. The patent information currently registered in the US FDA drug database is shown in Table 2 below. There are a total of 9 US patents, which correspond to 2 patent families, and 5 corresponding CN patents. It can be inferred that UCB has comprehensive patent protection for Zilucoplan, making it difficult for companies to imitate and challenge Zilucoplan.

Table 2

References:

1. https://mp.weixin.qq.com/s/fQye_4fW2qReEE8qwfXBxg

2. https://health.ifeng.com/c/8nMlPrdKYu9