{"title":"Oral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria.","authors":"Bo Xu,Jiecan Zhou","doi":"10.1080/17512433.2024.2403638","DOIUrl":null,"url":null,"abstract":"INTRODUCTION\r\nParoxysmal 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™), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on March 29, 2024. The primary purpose of this review is to examine the clinical efficacy and safety of danicopan.\r\n\r\nAREAS COVERED\r\nWe systematically searched for articles on PubMed, Web of Science, and three publishers Springer, Elsevier, Wiley up to May 6, 2024.\r\n\r\nEXPERT OPINION\r\nDanicopan 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.","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":"7 1","pages":""},"PeriodicalIF":3.6000,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Review of Clinical Pharmacology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/17512433.2024.2403638","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
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™), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on March 29, 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 May 6, 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.
期刊介绍:
Advances in drug development technologies are yielding innovative new therapies, from potentially lifesaving medicines to lifestyle products. In recent years, however, the cost of developing new drugs has soared, and concerns over drug resistance and pharmacoeconomics have come to the fore. Adverse reactions experienced at the clinical trial level serve as a constant reminder of the importance of rigorous safety and toxicity testing. Furthermore the advent of pharmacogenomics and ‘individualized’ approaches to therapy will demand a fresh approach to drug evaluation and healthcare delivery.
Clinical Pharmacology provides an essential role in integrating the expertise of all of the specialists and players who are active in meeting such challenges in modern biomedical practice.