Prospect of first-ever targeted oral prophylactic treatment
Researchers from Charité – Universitätsmedizin Berlin have successfully tested a medicine that can treat hereditary angioedema, a rare genetic disease characterized by severe swelling of the skin and mucous membranes. When taken daily in tablet form, the medicine helps prevent severe attacks of hereditary angioedema. The results of this international study have been published in the eminent New England Journal of Medicine*.
Hereditary angioedema (HAE) is caused by a genetic deficiency that results in an unregulated activation of the kallikrein-kinin system. This leads to an overproduction of bradykinin, a tissue hormone that causes fluids to leak from blood vessels into tissues, causing swelling. If left untreated, the swelling associated with HAE has the potential to cause death by asphyxiation. Specifically for this study, the researchers developed a ‘kallikrein inhibitor’ – a drug that inhibits the kallikrein enzyme, thereby halting the production of bradykinin and preventing tissue swelling.
Each of the 75 patients enrolled in this international study received either the kallikrein inhibitor (at varying doses) or a placebo. The study’s findings suggest that daily administration of the drug prevents symptoms from developing. Currently, most patients wait for signs of swelling to appear before they use one of the on-demand treatments available, all of which are administered either intravenously or subcutaneously.
Summarizing the results of this research, Prof. Dr. Marcus Maurer, Director of Research at the Department of Dermatology, Venereology and Allergology, says: “Should this drug receive its marketing authorization, it would be the first time that patients have access to a prophylactic treatment in tablet form that is safe, simple to use, and highly effective in preventing swelling.” He adds: “As a next step, we will need to conduct a follow-up study to confirm our results, and the planning for such a study is already underway. We also want to gain a better understanding of the precise way in which kallikrein-mediated effects contribute to symptom development, and we hope to identify other diseases in which kallikrein plays a significant role.”
*Aygören-Pürsün E, et al. Oral Plasma Kallikrein Inhibitor for Prophylaxis in Hereditary Angioedema. New England Journal of Medicine 2018;379:352-62. DOI: 10.1056/NEJMoa1716995.
Prof. Dr. Marcus Maurer
Department of Dermatology, Venereology and Allergology
Charité – Universitätsmedizin Berlin
t: +49 30 450 518 043
Back to Overview