Orphan drugs for the treatment of hereditary angioedema

Abstract

Introduction: Hereditary angioedema (HAE) is a rare disorder with recurring edema formation in the subcutis and the submucosa. The growing understanding of its pathophysiology yielded a number of new orphan drugs with diverse targets and delivery routes. Because HAE is bradykinin-mediated, its pharmacotherapy focuses on inhibiting the release, or the receptor action, of this vasoactive peptide.

Areas covered: This summary is intended as a brief review of the disease and of the medicinal products (non-pathogenic and pathogenic medications) available for its therapy. It also attempts to outline the choices in its complex management, and to assist in delivering appropriate care with minimum delay. The primary objective of therapy is to prevent edema, as well as to relieve its symptoms. Nowadays, many innovative drugs are available; their efficacy and safety have been demonstrated in controlled clinical trials. C1-inhibitor concentrates, prepared from human plasma, or produced by recombinant technology, are used for supplementation. Kallikrein inhibitors block the release of bradykinin, whereas icatibant interferes with its binding to the bradykinin B2 receptor.

Expert opinion: The expansion of therapeutic alternatives allows individualized treatment supported by recent international guidelines and recommendations.

Keywords::

• C1 inhibitor
• hereditary angioedema
• orphan drugs
• treatment

Acknowledgement

The author wishes to thank Miss Dorottya Csuka PhD student for compiling the list of references to the literature.