An evaluation of sebetralstat as the first oral on-demand therapy for Hereditary Angioedema.

Introduction: Hereditary Angioedema (HAE) is a rare genetic disease characterized by sudden episodes of edema that do not respond to conventional treatments. Modern therapies targeting the kallikrein-kinin system aim to alleviate HAE attacks, reduce their frequency and severity, and enhance patients' quality of life. Sebetralstat, a novel oral plasma kallikrein inhibitor, offers a promising option for rapid and effective on-demand treatment of HAE attacks.

Areas covered: This article evaluates the pharmacokinetics, pharmacodynamics, efficacy, and tolerability of sebetralstat based on developmental studies. We reviewed and analyzed seven pertinent original studies focusing on sebetralstat trials.

Expert opinion: Oral sebetralstat provides a convenient alternative to injectable treatments, allowing patients to take the drug at the first sign of an attack, improving management of acute symptoms by helping to resolve them quickly. Clinical trials demonstrated that sebetralstat exhibits rapid absorption, effective plasma kallikrein inhibition, and a favorable safety and efficacy profile. Its pharmacokinetic and pharmacodynamic properties suggest its potential for reliable control of HAE attacks.