HRS-5965

China's National Medical Products Administration (NMPA) has approved licancopan fumarate (HRS-5965) capsules, developed by Chengdu Suncadia Medicine Co., Ltd., a subsidiary of Jiangsu Hengrui Pharmaceuticals (HKEX: 01276), for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy. The approval, granted as a Class 1 innovative drug, marks the fourth oral Factor B inhibitor to reach the Chinese market for PNH, entering a rapidly consolidating alternative pathway inhibitor class. Licancopan fumarate (HRS-5965) capsules are approved at a 25 mg strength as a prescription drug. The compound is an oral, small-molecule inhibitor of complement Factor B, a serine protease central to the alternative complement pathway. By blocking Factor B, licancopan prevents assembly of the alternative pathway C3 convertase (C3bBb), suppressing both intravascular and extravascular hemolysis — a mechanistic distinction from terminal complement inhibitors such as eculizumab and ravulizumab, which target C5 and leave extravascular hemolysis incompletely controlled. Hengrui reported cumulative R&D investment in the licancopan program of approximately CNY 277.5 million (unaudited). The approval was supported by a randomized Phase III trial in 78 complement inhibitor-naïve PNH patients comparing oral licancopan with intravenous eculizumab over 24 weeks. Among 76 treated patients, 70.0% of those receiving licancopan achieved hemoglobin of at least 12 g/dL on at least three of four measurements between Weeks 18 and 24 without red blood cell transfusion, compared with 11.1% receiving eculizumab (P<0.0001). A hemoglobin increase of at least 2 g/dL was achieved by 95.0% versus 55.6%, respectively (P<0.0001), while all licancopan-treated patients remained transfusion-free after Week 2 versus 86.1% with eculizumab. The results were presented at EHA 2026. Treatment-related adverse events occurred in 72.5% of licancopan-treated patients versus 55.6% with eculizumab, with treatment-related serious adverse events reported in two licancopan patients; no major adverse vascular events occurred. The magnitude of the hemoglobin difference is consistent with the mechanistic advantage of proximal Factor B inhibition over C5 blockade. While eculizumab suppresses terminal complement-mediated intravascular hemolysis, upstream Factor B inhibition can additionally prevent C3-mediated extravascular destruction of red blood cells, a persistent source of anemia in some patients receiving C5 inhibitors. The approval places licancopan into direct competition with three already-approved oral Factor B inhibitors in China. Novartis' iptacopan hydrochloride (Fabhalta) was approved by the NMPA in 2024 and received US FDA approval in December 2023; Fabhalta recorded global sales of approximately USD 505 million in 2025, according to EvaluatePharma data cited by Hengrui. Lanoracopan hydrochloride (Yishining) from Createrna and ciprocopan succinate (Youkepan) from Haisco Pharmaceutical Group both received NMPA approval in 2026, with ciprocopan's approval — for the identical complement inhibitor-naïve PNH population — granted in July 2026. A separate marketing authorization application for licancopan covering PNH patients who remain anemic after prior treatment with C5 complement inhibitors is currently under NMPA review, which would extend the drug's addressable population to the treatment-experienced setting. PNH is classified within China's national rare disease list. The condition arises from a somatic mutation causing absence of GPI-anchored complement regulators CD55 and CD59 on erythrocyte surfaces, rendering cells susceptible to complement-mediated destruction. The clinical burden — including anemia, fatigue, thrombosis, and organ damage — has driven substantial investment in oral alternatives to the established IV biologic standard of care. Access the AllSci platform to explore the science behind the news. Spot something wrong? Report an issue with this article