Factor IX stimulants in preclinical and early phase trials for hemophilia B treatment.

IF 4.9 2区医学 Q1 PHARMACOLOGY & PHARMACY

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-11 DOI:10.1080/13543784.2024.2388565

Massimo Franchini, Daniele Focosi

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引用次数: 0

Abstract

Introduction: Hemophilia B is a X-linked rare inherited bleeding disorder characterized by coagulation factor IX (FIX) deficiency. Therapy for hemophilia B is aimed at replacing the FIX deficiency by means of several plasma-derived or recombinant FIX products. The recent availability of recombinant FIX concentrates with a prolonged FIX half-life represented a great technological advance, permitting more spaced drug infusions and reducing treatment burden among hemophilia B patients.

Areas covered: This review summarizes the main preclinical and phase 1/2 studies investigating the innovative hemostatic products for hemophilia B replacement therapy.

Expert opinion: The significant recent technological advantages in the treatment of hemophilia B has led to the development of innovative FIX products aimed at further extending FIX half-life and using increasingly effective and convenient modes of administration. These novel hemostatic agents, currently in the preclinical or early clinical phase of development, carry the potential of improving patients' health status and quality of life. Continuous research is anyway needed to offer such patients a concrete chance of conducting a normal existence, like to non-affected age-matched individuals.

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用于 B 型血友病治疗的临床前和早期试验中的因子 IX 促效剂。

简介血友病 B 是一种以凝血因子 IX（FIX）缺乏为特征的 X 连锁罕见遗传性出血性疾病。治疗 B 型血友病的目的是通过几种血浆衍生或重组 FIX 产品来替代 FIX 缺乏症。最近，具有延长 FIX 半衰期的重组 FIX 浓缩物的问世标志着一项巨大的技术进步，它允许更多的药物输注间隔，减轻了 B 型血友病患者的治疗负担：本综述概述了用于 B 型血友病替代疗法的创新止血产品的主要临床前研究和 1/2 期研究：最近在治疗 B 型血友病方面取得的巨大技术优势推动了创新型 FIX 产品的开发，这些产品旨在进一步延长 FIX 的半衰期，并采用越来越有效和方便的给药方式。这些新型止血剂目前正处于临床前或早期临床开发阶段，有望改善患者的健康状况和生活质量。无论如何，我们都需要继续开展研究，以便为这类患者提供与未受影响的同龄人一样正常生活的具体机会。

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 opinion on investigational drugs 医学-药学

CiteScore

10.00

自引率

0.00%

发文量

审稿时长

6-12 weeks

期刊介绍： Expert Opinion on Investigational Drugs (ISSN 1354-3784 [print], 1744-7658 [electronic]) is a MEDLINE-indexed, peer-reviewed, international journal publishing review articles and original papers on drugs in preclinical and early stage clinical development, providing expert opinion on the scope for future development. The Editors welcome: Reviews covering preclinical through to Phase II data on drugs or drug classes for specific indications, and their potential impact on future treatment strategies Drug Evaluations reviewing the clinical and pharmacological data on a particular drug Original Research papers reporting the results of clinical investigations on agents that are in Phase I and II clinical trials The audience consists of scientists, managers and decision-makers in the pharmaceutical industry, and others closely involved in R&D.