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New drug for rapid, on-demand self-medication in hereditary angioedema

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The active ingredient sebetralstat could replace current therapy forms

In hereditary angioedema (HAE), swelling occurs in one or more parts of the body without an identifiable trigger. Especially if the upper respiratory tract is affected, an HAE attack can be life-threatening. Active substances for on-demand medication are currently only available in the form of injections and infusions. In a Phase 2 study led by the University Hospital Frankfurt and the Charité - Universitätsmedizin Berlin, it has now been shown for the first time that an orally administered substance is effective. The therapy could replace the much more costly and stressful injections.

Patients with HAE have an alteration in their genetic makeup. The genetic disease manifests itself predominantly in childhood and adolescence and remains symptomatic in most affected individuals throughout their lives. It manifests itself as severe, localized swelling of the skin and mucous membranes, which can occur in various parts of the body. If the upper airways are involved, an HAE attack can be life-threatening. For example, if edema develops in the larynx as a result, this is one of the most common causes of death in HAE patients. Treatment guidelines therefore recommend treating the attacks as early as possible. Studies have shown that treating patients quickly significantly reduces the time to symptom relief and the overall duration of the attack.

HAE attacks can develop over several hours or occur quite suddenly. The frequency varies as much as the duration. In most cases, no direct trigger for the attacks can be identified. Various causes are suspected, including infections, medication, psychological stress situations, operations, trauma and changes in hormone balance. It is also difficult to predict when and on which part of the body the next attack will occur. However, with timely on-demand medication, hereditary angioedema is easily treatable, so that patients' lives are not endangered by unexpected attacks.


Oral on-demand therapy with sebetralstat
Currently approved therapies for HAE consist of on-demand and prophylactic medications, i.e. substance for acute treatment or prevention of attacks. However, administration has been burdensome to date: patients receive the substance through infusions or injections. These methods require training, they cause time delays in treatment, and can lead to undesirable side effects such as pain and hypersensitivity reactions at the infusion or injection site. The drug, now administered orally, avoided these serious side effects. Sebetralstat achieved faster symptom relief compared to placebo, reduced the severity of attacks, and significantly prolonged the time to conventional treatment. It is rapidly absorbed by the body and reaches maximum plasma concentrations within one hour, which is essential for symptom relief.

Based on the positive results of the study, a Phase 3 clinical trial with sebetralstat is currently being conducted, in which the on-demand treatment with the active ingredient will be re-evaluated.

Aygören-Pürsün E, Zanichelli A, Cohn DM, Cancian M, Hakl R, Kinaciyan T, Magerl M, Martinez-Saguer I, Stobiecki M, Farkas H, Kiani-Alikhan S, Grivcheva-Panovska V, Bernstein JA, Li HH, Longhurst HJ, Audhya PK, Smith MD, Yea CM, Maetzel A, Lee DK, Feener EP, Gower R, Lumry WR, Banerji A, Riedl MA, Maurer M. An investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema: a two-part, randomised, double-blind, placebo-controlled, crossover phase 2 trial. Lancet. 2023 Feb 11;401(10375):458-469. doi: 10.1016/S0140-6736(22)02406-0. PMID: 36774155.


official German press release


Emel Aygören-Pürsün, MD
Angioedema Outpatient Clinic
Clinic for Pediatrics and Adolescent Medicine
University Hospital Frankfurt


Marcus Maurer, MD
Executive Director of the Institute of Allergology
Charité - Universitätsmedizin Berlin

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