Beijing News (Reporter Wang Kala) According to the PharmaCube Global Approved New Drug Database, Novartis' innovative rare disease drug Iptacopan Hydrochloride Capsules was approved for marketing by the National Medical Products Administration on April 26, just four months after the U.S. Food and Drug Administration (FDA) approved the drug. The indication approved in China is for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy.
The approval of Iptacopan Hydrochloride Capsules in China is based on the results of the pivotal Phase III APPOINT-PNH global and China subgroup clinical study (with 50% of participants enrolled from China). The study demonstrated the efficacy, safety, and tolerability of Iptacopan Hydrochloride Capsules in adult PNH patients who had not previously received complement inhibitor therapy. After 24 weeks of treatment, most patients achieved hemoglobin levels of 12 g/dL or higher, nearly all patients avoided the need for transfusions, and patient-reported fatigue also improved.
PNH was included in the first batch of the rare disease catalog in 2018. According to the "Chinese Expert Consensus on Clonal Screening and Complement Inhibitor Treatment Monitoring for Paroxysmal Nocturnal Hemoglobinuria (2024 Edition)," PNH is a rare acquired disorder caused by somatic mutations in the glycosylphosphatidylinositol class A (PIGA) gene at the level of pluripotent hematopoietic stem cells. It is primarily characterized by recurrent intravascular hemolysis, a tendency for thrombosis, and varying degrees of bone marrow failure. Although PNH is a benign condition, under traditional treatment methods, the 10-year overall survival (OS) rate of patients is only 70.7%-77.6%, with both survival rates and quality of life urgently needing improvement.
As a chronic, progressive, and life-threatening rare blood disorder, PNH can occur at any age but is more commonly seen in people aged 30-40, imposing a lifelong heavy burden on young and middle-aged adults. The current standard treatment for PNH is anti-complement C5 therapy. However, after receiving this treatment, a significant number of patients still experience residual anemia, fatigue, and transfusion dependency.
Iptacopan Hydrochloride Capsules are the world's first specific oral inhibitor of complement factor B, acting on the proximal pathway of the alternative complement pathway in the immune system. It can comprehensively control intravascular hemolysis and extravascular hemolysis, making up for the shortcomings of anti-C5 antibody therapy. Information released by the PNH Patient Home WeChat Official Account shows that, as the first oral monotherapy for adult PNH treatment, Iptacopan Hydrochloride Capsules can improve treatment adherence, enhance medication experience, and improve quality of life.
Proofread by Lu Qian