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.

New drugs for the treatment of hereditary angioedema / G. Costanzo, G. Sambugaro, S. Sartorio, A. Zanichelli, D. Firinu. - In: EXPERT OPINION ON BIOLOGICAL THERAPY. - ISSN 1471-2598. - 25:1(2025 Jan), pp. 79-91. [10.1080/14712598.2024.2441845]

New drugs for the treatment of hereditary angioedema

S. Sartorio;A. Zanichelli
Penultimo
;
2025

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.
hereditary angioedema; kallikrein/kinin system; bradykinin; C1-inhibitor; biological drugs
Settore MEDS-05/A - Medicina interna
gen-2025
14-dic-2024
Article (author)
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/1194975
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