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ABSTRACT
Introduction
Hereditary angioedema due to C1-inhibitor (C1-INH-HAE) is a rare disease characterized by unpredictable swelling attacks that may be life-threatening when affecting the upper airways. Understanding the pathophysiology of HAE and the mechanism of bradykinin-mediated angioedema allowed the development of new therapies for the treatment of HAE: clinical trials are ongoing to expand the number of drugs available for on-demand treatment and prophylaxis.
Areas covered
Authors discuss the products that have been used to treat this disease for many years and present the most recently marketed products and those which are under development.
Expert opinion
Significant therapeutic progress has been made in HAE. In particular, drugs targeting specific molecules involved in the angioedema formation were developed and studies with new drugs are ongoing. In the coming years, more effective therapies with easier administration route options for on-demand treatment and long-term prophylaxis will be available to treat this disease and the variety of patients. Gene therapy strategies may offer a definitive treatment. High costs of current and new drugs may be a limiting factor for their availability, especially in developing countries.
Acknowledgments
We acknowledge Laura Fascio Pecetto from SEEd Medical Publishers that provided medical writing assistance, publishing support, and journal styling services.
Disclosure statement
A Zanichelli received speaker/consultancy fees and/or was a member of medical/advisory boards for BioChrist, CSL Behring, Kallvista, Pharming, Pharvaris and Takeda. M Triggiani received speaker/consultancy fees and/or was a member of medical/advisory boards for BioChrist and Takeda. F Arcoleo received speaker/consultancy fees by CSL Behring and Takeda. M Cancian received travel grants from CSL Behring, Menarini, Novartis, Shire-Takeda and consultancy fees from Biocryst, CSL Behring, Shire-Takeda. His Institution received scientific grants from CSL Behring and Shire-Takeda. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Reviewer disclosures
A reviewer on this manuscript has received honorarium from CSL-Behring, Takeda and Torii, consultant fee from Takeda, BioCryst and Pharvaris. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.
List of abbreviations
| C1-INH | = | C1-inhibitor |
| C1-INH-HAE | = | Hereditary angioedema due to C1-inhibitor |
| HAE | = | hereditary angioedema |
| KKS | = | kallikrein-kinin system |
| LTP | = | long-term prophylaxis |
| pdC1-INH | = | plasma-derived C1-inhibitor |
| PICC | = | peripherally inserted central catheter |
| STP | = | short-term prophylaxis |