Efanesoctocog alfa for the prevention and treatment of bleeding in patients with hemophilia A.

IF 2.3 4区医学 Q2 HEMATOLOGY

Expert Review of Hematology Pub Date : 2023-07-01 Epub Date: 2023-06-15 DOI:10.1080/17474086.2023.2223925

Barbara A Konkle

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引用次数: 1

Abstract

Introduction: Hemophilia A is an inherited bleeding disorder due to a deficiency of coagulation factor VIII (FVIII). Prevention and treatment of bleeding is traditionally through intravenous infusion of a FVIII concentrate. Modifications of recombinant FVIII (rFVIII) with an aim to prolong the half-life have been modest, thought because FVIII is dependent on plasma von Willebrand factor (VWF) for its half-life. Efanesoctocog alfa (ALTUVIIIO), approved by the Federal Drug Administration (FDA) in February 2023, was made independent of endogenous VWF by linking of the FVIII-binding D'D3 domain of VWF to B-domain deleted single chain FVIII.

Areas covered: This review will outline the development of efanesoctocog alfa and the pharmacokinetic and safety data from clinical trials, as well as efficacy data from the phase 3 trials. These data formed the basis for the FDA approval.

Expert opinion: Efanesoctocog alfa is a new type of FVIII replacement with an extended half-life allowing once weekly dosing to achieve hemostasis and FVIII trough levels of 13-15 IU/dL. This provides a highly effective option for treatment and prevention of bleeding in hemophilia A, where FVIII levels are easily measured. It also provides an option for treatment of bleeding and coverage for surgery with few infusions.

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埃法尼酮预防和治疗血友病A患者出血。

简介：血友病A是一种由于凝血因子VIII（FVIII）缺乏引起的遗传性出血性疾病。出血的预防和治疗传统上是通过静脉输注FVIII浓缩物。以延长半衰期为目的的重组FVIII（rFVIII）的修饰是适度的，这被认为是因为FVIII的半衰期依赖于血浆血管性血友病因子（VWF）。埃法尼司他康（ALTUVIIIO）于2023年2月获得美国联邦药品监督管理局（FDA）批准，通过将VWF的FVIII结合D’D3结构域与B结构域缺失的单链FVIII连接，使其独立于内源性VWF。涵盖的领域：本综述将概述埃法尼司他康的开发以及临床试验的药代动力学和安全性数据，以及来自3期试验的疗效数据。这些数据构成了美国食品药品监督管理局批准的基础。专家意见：Efanesoctocog alfa是一种新型的FVIII替代品，半衰期延长，每周给药一次，可达到止血效果，FVIII谷值为13-15 IU/dL。这为治疗和预防血友病a出血提供了一种非常有效的选择，其中FVIII水平很容易测量。它还提供了一种治疗出血的选择，并为很少输液的手术提供了覆盖范围。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Expert Review of Hematology HEMATOLOGY-

CiteScore

4.70

自引率

3.60%

发文量

审稿时长

6-12 weeks

期刊介绍： Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.