Haisco Secures NMPA Approval for First-in-Class PNH Oral Therapy

Paroxysmal Nocturnal Hemoglobinuria (PNH) involves a chronic, life-altering breakdown of red blood cells, often leading to severe fatigue, thrombosis, and renal impairment. While existing C5 inhibitors address some symptoms, they frequently fail to block upstream complement activation, leaving many patients reliant on transfusions. Haisco’s new treatment, identified in clinical development as HSK39297, targets the alternative complement pathway to suppress the amplification loop directly.

Clinical data indicates that the therapy offers a robust safety profile with no dosage adjustments required for patients with hepatic or renal impairment. In Phase III trials, 94.6% of patients achieved transfusion-free status, with hemoglobin levels rising significantly. Beyond domestic approval, Haisco has moved to capitalize on its intellectual property by licensing global development and commercialization rights—excluding Greater China, Southeast Asia, and India—to U.S.-based Nuvectis Pharma. This deal, valued at over $1.4 billion in potential milestones, underscores the growing international footprint of China’s pharmaceutical innovation sector.