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Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety

Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disord...

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Autores principales: Lunn, Michael, Santos, Carah, Craig, Timothy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2010
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3262319/
https://www.ncbi.nlm.nih.gov/pubmed/22282695
http://dx.doi.org/10.2147/JBM.S9576
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author Lunn, Michael
Santos, Carah
Craig, Timothy
author_facet Lunn, Michael
Santos, Carah
Craig, Timothy
author_sort Lunn, Michael
collection PubMed
description Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disorder. The generation of an HAE attack is caused by the depletion and/or consumption of C1-inhibitor manifested as subcutaneous or submucosal edema of the upper airway, face, extremities, or gastrointestinal tract. Attacks can be severe and potentially life-threatening, particularly with laryngeal involvement. Despite the availability of C1-INH for the treatment of HAE since the 1980s in Europe and other countries, HAE treatment in the United States was limited to androgen therapy. The human plasma-derived C1 esterase inhibitor (Cinryze™), distributed by Lev Pharmaceuticals, was approved in October 2008 for the prevention of HAE attacks based on the results of a phase III clinical trial. This review aims to describe the history of C1-INH replacement in HAE as well as the pharmacology, efficacy and safety of C1-INH, concentrating on Cinryze as the first approved chronic replacement treatment for the prophylaxis of HAE attacks.
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spelling pubmed-32623192012-01-26 Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety Lunn, Michael Santos, Carah Craig, Timothy J Blood Med Review Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disorder. The generation of an HAE attack is caused by the depletion and/or consumption of C1-inhibitor manifested as subcutaneous or submucosal edema of the upper airway, face, extremities, or gastrointestinal tract. Attacks can be severe and potentially life-threatening, particularly with laryngeal involvement. Despite the availability of C1-INH for the treatment of HAE since the 1980s in Europe and other countries, HAE treatment in the United States was limited to androgen therapy. The human plasma-derived C1 esterase inhibitor (Cinryze™), distributed by Lev Pharmaceuticals, was approved in October 2008 for the prevention of HAE attacks based on the results of a phase III clinical trial. This review aims to describe the history of C1-INH replacement in HAE as well as the pharmacology, efficacy and safety of C1-INH, concentrating on Cinryze as the first approved chronic replacement treatment for the prophylaxis of HAE attacks. Dove Medical Press 2010-08-24 /pmc/articles/PMC3262319/ /pubmed/22282695 http://dx.doi.org/10.2147/JBM.S9576 Text en © 2010 Lunn et al, publisher and licensee Dove Medical Press Ltd. This is an Open Access article which permits unrestricted noncommercial use, provided the original work is properly cited.
spellingShingle Review
Lunn, Michael
Santos, Carah
Craig, Timothy
Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
title Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
title_full Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
title_fullStr Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
title_full_unstemmed Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
title_short Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
title_sort cinryze(™) as the first approved c1 inhibitor in the usa for the treatment of hereditary angioedema: approval, efficacy and safety
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3262319/
https://www.ncbi.nlm.nih.gov/pubmed/22282695
http://dx.doi.org/10.2147/JBM.S9576
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