UCB’s zilucoplan, world’s first subcutaneous C5 inhibitor for gMG, approved in China

On October 11, biopharmaceutical company UCB announced that China's National Medical Products Administration (NMPA) has approved the marketing authorization for zilucoplan (marketed in China under the brand name 卓倍可®). The therapy is indicated as an add-on to standard treatment for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

Reportedly, zilucoplan is the world's first and only next-generation C5 complement inhibitor for AChR antibody-positive gMG that can be administered subcutaneously and is suitable for self-administration. It also features a dual mechanism of action. As an innovative therapy with this dual inhibitory mechanism, it effectively blocks complement-mediated damage to the neuromuscular junction. Patients can complete the subcutaneous injection at home in just 5–8 seconds.

According to data, generalized myasthenia gravis (gMG) is a rare, chronic, and heterogeneous autoimmune disease characterized primarily by dysfunction at the neuromuscular junction (NMJ). A 2025 Frost & Sullivan report estimates approximately 1.2 million gMG patients globally, with about 220,000 residing in China. The common pathogenic antibodies in gMG mainly include anti-acetylcholine receptor (AChR) antibodies and anti-muscle-specific tyrosine kinase (MuSK) antibodies. Among these, AChR antibodies are the most prevalent, detectable in approximately 80–90% of patients.

In AChR antibody-positive patients, these abnormal antibodies activate the complement system within the immune system. This process ultimately leads to the formation of the Membrane Attack Complex (MAC) at the connection between nerves and muscles. The MAC damages the neuromuscular junction, resulting in muscle weakness and impaired signal transmission. Preventing the formation of this complex can mitigate damage, help preserve neural signal transmission, and protect muscle function.

The approval of zilucoplan is based on the Phase 3 RAISE study (NCT04115293), with the research findings published in The Lancet Neurology in May 2023. The study demonstrated that in AChR antibody-positive gMG patients, the treatment resulted in statistically significant improvements in both the MG-ADL and QMG scores compared to placebo, with a favorable safety profile. Trial results indicated that patients experienced significant improvements in functions such as breathing, swallowing, and speaking within the first week of treatment. Long-term therapy provided sustained and stable efficacy and reduced the reliance on intravenous immunoglobulin (IVIg) and plasma exchange (PE).

This approval enables UCB to simultaneously offer two innovative therapies—zilucoplan and rozanolixizumab—in China, establishing the company as the only biopharmaceutical firm worldwide capable of providing two distinct targeted treatment pathways.