Oral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria

被引:0
作者
Xu, Bo [1 ,2 ,3 ,4 ,5 ]
Zhou, Jiecan [1 ,2 ,3 ,4 ,5 ]
机构
[1] Univ South China, Sch Pharmaceut Sci, Hengyang Med Sch, Hengyang, Hunan, Peoples R China
[2] Univ South China, Affiliated Hosp 1, Hunan Prov Clin Med Res Ctr Drug Evaluat Major Chr, Hengyang Med Sch, Hengyang 421001, Hunan, Peoples R China
[3] Univ South China, Affiliated Hosp 1, Hengyang Clin Pharmacol Res Ctr, Hengyang Med Sch, Hengyang, Hunan, Peoples R China
[4] Univ South China, Affiliated Hosp 1, Hengyang Med Sch, Hengyang Key Lab Clin Pharmacol, Hengyang, Hunan, Peoples R China
[5] Univ South China, Affiliated Hosp 1, Hengyang Med Sch, Pharm Dept, Hengyang, Hunan, Peoples R China
关键词
Danicopan; Voydeya; paroxysmal nocturnal hemoglobinuria; extravascular hemolysis; ACH-4471; ECULIZUMAB; HEMOLYSIS; PNH;
D O I
10.1080/17512433.2024.2403638
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by episodic hemolysis, with additional clinical manifestations including thrombosis and bone marrow failure. The US FDA approved a complement factor D inhibitor, danicopan (Voydeya (TM)), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on 29 March 2024. The primary purpose of this review is to examine the clinical efficacy and safety of danicopan. Areas covered: We systematically searched for articles on PubMed, Web of Science, and three publishers Springer, Elsevier, Wiley up to 6 May 2024. Expert opinion: Danicopan acts on the alternative pathway of the complement cascade, preferentially controlling C3 fragment-mediated extravascular hemolysis. Recommended dosage is 150 mg orally three times a day, which can be increased to 200 mg three times a day when necessary. Vaccination is required before administration to prevent infections by encapsulated bacteria. In a pivotal phase 3 trial ALPHA, danicopan significantly increased hemoglobin levels compared to placebo (p < 0.0001), 60% of patients experienced an increase in hemoglobin levels of at least 2 g/dL, compared to none in the placebo group (adjusted difference of 47%; p < 0.0001). Common adverse events during danicopan treatment include headache and upper respiratory tract infection.
引用
收藏
页码:857 / 864
页数:8
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