April 26,NovartisAnnouncing its hydrochlorideEpicopanCapsule (iptacopan) has been approved for marketing in China, indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy.Iptacopan is a first-in-class, oral inhibitor targeting the complement alternative pathway B factor developed by Novartis. The product acts upstream of the terminal C5 pathway, controlling both intravascular and extravascular hemolysis, addressing the limitations of anti-C5 antibodies while providing patients with an oral monotherapy option.In December 2023, Iptacopan received FDA approval for the treatment of adult PNH patients, becoming the first orally administered monotherapy for adult PNH to be approved by the FDA. Just four months after receiving FDA approval, Iptacopan was successfully approved in China, marking another example of Novartis driving the accelerated entry of innovative global drugs into China with "China speed."The approval in China was 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 ipocaplon in adult PNH patients who had not previously received complement inhibitor therapy. After 24 weeks of treatment, the majority of patients achieved hemoglobin levels of 12 g/dL or higher, nearly all patients avoided the need for transfusions, and patient-reported fatigue also improved.Paroxysmal Nocturnal Hemoglobinuria (PNH) is a complement-mediated chronic rare blood disorder. According to statistics, the incidence of PNH is about one to two per million. The incidence rate in Asian populations is higher than in Western countries and can occur at any age, commonly between 30-40 years old. In PNH patients, mutations in the PIG-A gene lead to the absence of crucial complement regulatory proteins CD55 and CD59 on the surface of blood cells. Consequently, cells are more susceptible to complement activation. Clinically, it mainly manifests as hemolysis, bone marrow failure, and thrombosis.Anti-complement C5 therapy(EculizumabOrRavulizumab) was previously the internationally recognized standard treatment for PNH, but after anti-C5 therapy, a majority of patients still experience residual anemia, fatigue, and transfusion dependence, significantly impacting their quality of life.Copyright © 2024 PHARMCUBE. All Rights Reserved.
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