The therapeutic landscape for hereditary angioedema: more change on the horizon?

Abstract

Hereditary angioedema (HAE) is a rare immunologic condition that has received significant attention within the field of allergy and immunology in recent years. Individuals affected by HAE often have tremendous diagnostic delays, inappropriate and ineffective therapy, substantial morbidity, and considerable risk of death from asphyxiation. [1] Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013; 111: 329-336 Abstract Full Text Full Text PDF PubMed Scopus (76) Google Scholar Medical management of HAE in the United States was seriously hampered by a lack of Food and Drug Administration–approved HAE acute therapy until 2009. Since then, the treatment paradigm for HAE has rapidly evolved. Advances in understanding the pathophysiology of HAE eventually led to the clinical development of medications addressing the underlying C1 esterase inhibitor (C1INH) deficiency and the resulting dysregulation of the contact (kallikrein–bradykinin) system. [2] Kaplan A.P. Joseph K. The bradykinin-forming cascade and its role in hereditary angioedema. Ann Allergy Asthma Immunol. 2010; 104: 193-204 Abstract Full Text Full Text PDF PubMed Scopus (114) Google Scholar After rigorous clinical trials, C1INH concentrates, ecallantide, and icatibant are now licensed HAE therapies in the United States and other parts of the world. [3] Parikh N. Riedl M.A. New therapeutics in C1INH deficiency: a review of recent studies and advances. Curr Allergy Asthma Rep. 2011; 11: 300-308 Crossref PubMed Scopus (9) Google Scholar The availability of these treatments has dramatically altered HAE management and almost certainly improved patients' lives, although recent longitudinal outcomes data for HAE are sparse. A phase 1 study investigating DX-2930 in healthy subjectsAnnals of Allergy, Asthma & ImmunologyVol. 113Issue 4PreviewDX-2930 is a human monoclonal antibody inhibitor of plasma kallikrein under investigation for long-term prophylaxis of hereditary angioedema. Full-Text PDF Open Access