Ravulizumab-CWVZ

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

iStock, Nuthawut Somsuk AstraZeneca will push through with its other Phase 3 studies for volrustomig, including in cervical cancer and mesothelioma. AstraZeneca will discontinue a Phase 3 study for its investigational bispecific antibody volrustomig in non-small cell lung cancer following disappointing interim data. The pharma was testing volrustomig in the late-stage eVOLVE-Lung02 trial in combination with chemotherapy as a first-line treatment option for patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have PD-L1 expression levels lower than 50%. The study compared the volrustomig regimen against Merck’s Keytruda plus chemotherapy. A planned review of the trial data showed that volrustomig was unlikely to hit either of the study’s primary endpoints of progression-free survival or overall survival, according to a Monday morning release . Thus, the independent data panel recommended the study’s termination. AstraZeneca did not provide specific data in its release. The pharma is “disappointed” with the results, Susan Galbraith, executive vice president of Oncology Hematology R&D, said in a prepared statement, adding that the company “will learn from this trial.” AstraZeneca will continue its other Phase 3 volrustomig studies as planned in mesothelioma, cervical cancer, and head and neck squamous cell carcinoma. The latest blow continues what has been a rough few months for AstraZeneca in the clinic. Most notably, the pharma last month announced that its antisense therapy Wainua, which it shares with Ionis Pharmaceuticals, failed to show significant cardiovascular benefit in a Phase 3 study transthyretin amyloidosis-cardiomyopathy (ATTR-CM) study. The stumble sent waves across the ATTR-CM space, casting doubt on the silencer class of therapies—which work by suppressing transthyretin expression—and giving a boost to stabilizers, which prevent the protein from misfolding, a crucial pathogenic step. Cardiovascular disease AstraZeneca, Ionis’ Wainua woes send waves across ATTR-CM space The failure of AstraZeneca and Ionis’ Wainua in a late-stage study of ATTR-CM casts doubt on Alnylam’s next-generation candidate but is good news for others in the space, including BridgeBio and Intellia Therapeutics. July 10, 2026 · 3 min read · Tristan Manalac Read more Aside from Wainua, AstraZeneca also revealed in July that its rare disease drug Ultomiris failed to achieve statistically significant event-free survival in the Phase 3 ALXN1210-TMA-313 trial, which enrolled patients 12 years and older with thrombotic microangiopathy who had received hematopoietic stem cell transplant. That same day, during its second quarter earnings report , the pharma disclosed some other clinical disappointments, including in the Phase 3 CLARITY-Gastric01 trial, where its antibody-drug conjugate sonesitatug vedotin failed to significantly boost progression-free survival in the overall study population of patients with advanced or metastatic gastric or gastroesophageal junction adenocarcinoma. The asset did improve overall survival in these patients. Earnings AstraZeneca has no plans to ramp up dealmaking in wake of Wainua failure AstraZeneca remains “very much on track” to hit its 2030 goal of reaching $80 billion in revenue despite the recent late-stage failure of Wainua in ATTR-cardiomyopathy, CEO Pascal Soriot said during the company’s Q2 earnings presentation on Monday. July 27, 2026 · 3 min read · Tristan Manalac Read more Even in the case of a clinical win, AstraZeneca can’t seem to catch a break. In June, its investigational GLP-1 pill elecoglipron demonstrated a nearly 10% weight reduction at 25 weeks as compared with placebo. At 36 weeks, placebo-adjusted weight loss increased to 11.5%. While BMO Capital Markets at the time said these data pointed to elecoglipron’s “promising efficacy,” the firm added that the results were otherwise “relatively underwhelming,” especially compared with other drugs in development—particularly Structure Therapeutics’ aleniglipron, which showed a 15.3% placebo-adjusted weight reduction at 36 weeks . Editor’s note (Aug. 18): The headline of this story was changed to more accurately reflect AstraZeneca’s recent clinical developments.