Investigational drugs in phase I and phase II clinical trials for hereditary angioedema

H. Farkas, Márta Lídia Debreczeni, Kinga Viktória Kőhalmi

Research output: Contribution to journalReview article

4 Citations (Scopus)

Abstract

Introduction: Hereditary angioedema (HAE) with C1-inhibitor deficiency (C1-INH-HAE) is a rare bradykinin-mediated disease characterized by recurrent subcutaneous and/or submucosal angioedematous attacks (HAE attacks), which occur unpredictably. The recurrent HAE attacks do not respond to conventional treatments, and may evolve into a life-threatening condition; therefore, special therapy is required. Areas covered: The agents used so far for the acute management of HAE attacks act by blocking the release of bradykinin, or its binding to its receptor. By contrast, the investigational medicinal products under evaluation in Phase I and II clinical trials are targeted at the prevention of HAE attacks. Chemically, these new drugs are small synthetic molecules, oligonucleotides, or antibodies, which inhibit either kallikrein, or Factor XII. Expert opinion: The key considerations for the development of new medicinal products include more straightforward dosing, self-administration, longer duration of action, and keeping the patient attack-free. This review summarizes the status and the findings of the currently ongoing Phase I and Phase II clinical trials of C1-INH-HAE.

Original languageEnglish
Pages (from-to)87-103
Number of pages17
JournalExpert Opinion on Investigational Drugs
Volume27
Issue number1
DOIs
Publication statusPublished - Jan 2 2018

Fingerprint

Investigational Drugs
Hereditary Angioedemas
Phase II Clinical Trials
Bradykinin
Factor XII
Clinical Trials, Phase I
Kallikreins
Self Administration
Expert Testimony
Oligonucleotides
Antibodies

Keywords

  • acute treatment
  • C1-inhibitor deficiency
  • Factor XII inhibitor
  • Hereditary angioedema
  • intravenous plasma-derived C1-inhibitor
  • kallikrein inhibitor
  • prophylaxis
  • recombinant human C1-inhibitor
  • subcutaneous plasma-derived C1-inhibitor

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

Cite this

Investigational drugs in phase I and phase II clinical trials for hereditary angioedema. / Farkas, H.; Debreczeni, Márta Lídia; Kőhalmi, Kinga Viktória.

In: Expert Opinion on Investigational Drugs, Vol. 27, No. 1, 02.01.2018, p. 87-103.

Research output: Contribution to journalReview article

Farkas, H. ; Debreczeni, Márta Lídia ; Kőhalmi, Kinga Viktória. / Investigational drugs in phase I and phase II clinical trials for hereditary angioedema. In: Expert Opinion on Investigational Drugs. 2018 ; Vol. 27, No. 1. pp. 87-103.
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