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.