恒瑞医药：富马酸立康可泮(HRS-5965)胶囊的药品上市许可申请获国家药监局受理

Core Viewpoint - Heng Rui Medicine's subsidiary Chengdu Shengdi Pharmaceutical has received a notice of acceptance from the National Medical Products Administration for the marketing authorization application of HRS-5965 capsules, a treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) [1][2] Group 1: Company Developments - The application for HRS-5965's market approval is based on a pivotal study involving PNH patients who had not previously received complement inhibitor treatment [1] - The clinical study (HRS-5965-301) was a Phase III trial comparing HRS-5965 capsules with Eculizumab in treating PNH patients, led by prominent researchers from the Chinese Academy of Medical Sciences [1] - The study included 76 patients across 13 centers nationwide, showing that HRS-5965 significantly improved hemoglobin levels, reduced the need for blood transfusions, and enhanced quality of life compared to Eculizumab [1] Group 2: Industry Context - PNH is a rare acquired hemolytic disease characterized by the deficiency of CD55 and CD59, leading to complement-mediated intravascular hemolysis, and has been included in the national rare disease directory due to its low incidence/prevalence [2] - HRS-5965 capsules act as a complement factor B inhibitor, which can suppress complement-mediated hemolysis and improve hemoglobin levels [2] - Currently, the only other drug targeting this indication is Novartis's Iptacopan (Fabhalta), which is projected to generate global sales of approximately $129 million in 2024 [2] - The cumulative R&D investment for HRS-5965 capsules has reached approximately 218 million yuan [2]