New drugs for the treatment of hereditary angioedema.

IF 3.6 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Expert Opinion on Biological Therapy Pub Date : 2025-01-01 Epub Date: 2024-12-14 DOI:10.1080/14712598.2024.2441845
Giulia Costanzo, Giada Sambugaro, Silvio Sartorio, Andrea Zanichelli, Davide Firinu
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引用次数: 0

Abstract

Introduction: Revolutionary drugs have been developed and approved in the last 5 years for the treatment of hereditary angioedema (HAE). Increased knowledge of HAE pathophysiology has led to the development of innovative drugs for self-administered on-demand therapy and for short- and long-term prophylaxis (LTP). This has rendered possible a personalized approach for patients, allowing greater control of symptoms, better quality of life and reduction in the incidence of adverse effects linked to old treatments.

Areas covered: In this review we have highlighted which treatments are currently approved for HAE and some of the promising future therapies under development.

Expert opinion: While the first generation of approved treatments improved disease control for most patients, innovative therapies may allow individualized action plans and reduce complexity of treatment. Switching therapies due to insufficient efficacy, patient preference or adverse events is becoming progressively feasible and common. New LTPs may lead to the achievement of attack-free remission, allowing us to hopefully reach complete disease control for all patients and further improving their quality of life. In particular, LTPs with longer administration intervals, and on-demand therapies administered via the oral route will have a key role and will set more prominent targets for the upcoming drugs.

治疗遗传性血管性水肿的新药。
在过去的5年里,革命性的药物已经被开发并批准用于治疗遗传性血管性水肿(HAE)。对HAE病理生理学知识的增加导致了创新药物的发展,用于自我给药和短期和长期预防(LTP)。这使得针对患者的个性化治疗成为可能,从而可以更好地控制症状,提高生活质量,减少与旧治疗方法相关的不良反应的发生率。涵盖领域:在本综述中,我们强调了目前已批准的治疗HAE的治疗方法以及正在开发的一些有希望的未来治疗方法。专家意见:虽然第一代批准的治疗方法改善了大多数患者的疾病控制,但创新疗法可能允许个性化的行动计划并降低治疗的复杂性。由于疗效不足、患者偏好或不良事件而转换治疗正逐渐变得可行和普遍。新的ltp可能会导致无发作缓解的实现,使我们有希望实现对所有患者的完全疾病控制,并进一步提高他们的生活质量。特别是,具有较长给药间隔的ltp和通过口服途径进行的按需治疗将发挥关键作用,并将为即将推出的药物设定更突出的靶点。
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来源期刊
Expert Opinion on Biological Therapy
Expert Opinion on Biological Therapy 医学-生物工程与应用微生物
CiteScore
8.60
自引率
0.00%
发文量
96
审稿时长
3-8 weeks
期刊介绍: Expert Opinion on Biological Therapy (1471-2598; 1744-7682) is a MEDLINE-indexed, international journal publishing peer-reviewed research across all aspects of biological therapy. Each article is structured to incorporate the author’s own expert opinion on the impact of the topic on research and clinical practice and the scope for future development. The audience consists of scientists and managers in the healthcare and biopharmaceutical industries and others closely involved in the development and application of biological therapies for the treatment of human disease. The journal welcomes: Reviews covering therapeutic antibodies and vaccines, peptides and proteins, gene therapies and gene transfer technologies, cell-based therapies and regenerative medicine Drug evaluations reviewing the clinical data on a particular biological agent Original research papers reporting the results of clinical investigations on biological agents and biotherapeutic-based studies with a strong link to clinical practice Comprehensive coverage in each review is complemented by the unique Expert Collection format and includes the following sections: Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results; Article Highlights – an executive summary of the author’s most critical points.
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