On January 19, AstraZenecaAstraZeneca announces the approval and launch of its first-in-class oral complement factor D inhibitor, Voydeya (danicopan), in Japan, achieving the world's first approval. It is used to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and can be combined with C5 inhibitor therapy when patients show an inadequate response to C5 inhibitors.To address the need for clinically significant extravascular hemolysis (EVH) in PNH patients (approximately 10-20%) receiving C5 inhibitor therapy。
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare and serious blood disorder characterized by the destruction of red blood cells within blood vessels (also known as intravascular hemolysis) and the activation of white blood cells and platelets, which can lead to thrombosis. In patients with PNH, the complement system is abnormally activated; therefore, treatment with a C5 complement inhibitor can be used. However, patients receiving C5 complement inhibitor therapy often experience clinically significant extravascular hemolysis (EVH).Danicopan is an oral medication used as an add-on to C5 inhibitor therapies Ultomiris or Soliris. The drug selectively inhibits complement factor D, which plays a key role in the amplification of the complement system response.The approval by Japan's Ministry of Health, Labour and Welfare (MHLW) was based on the positive results of the pivotal ALPHA Phase III trial. The results of the 12-week initial assessment period of the trial have been published in The Lancet Haematology.ALPHA is a randomized, placebo-controlled Phase III study, enrolling a total of 84 patients.Paroxysmal Nocturnal Hemoglobinuria Experiencing Significant Extravascular Hemolysis in Clinical PracticePatients. The results showed that, compared with the placebo group, the change in hemoglobin levels from baseline at 12 weeks in the Danicopan group was statistically significant and clinically meaningful, achieving the primary endpoint of the study. The study also met key secondary endpoints, including avoidance of transfusions and changes in FACIT fatigue scores.Overall, Danicopan demonstrated good safety and tolerability.It is worth mentioning that this drug was initially developed by Alexion. In 2020, AstraZeneca acquired the company for approximately $39 billion, gaining access to a variety of complement drugs and innovative therapies for rare diseases.Copyright © 2024 PHARMCUBE. All Rights Reserved.
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