DefinitionThis section has been translated automatically.
Sebetralstat is an investigational oral plasma kallikrein inhibitor for the acute treatment of hereditary angioedema (HAE).
Pharmacodynamics (Effect)This section has been translated automatically.
Sebetralstat works in the treatment of hereditary angioedema (HAE) by targeting the kallikrein-kinin system (KKS) cascade and selectively inhibiting the plasma kallikrein that triggers HAE attacks. Sebetralstat is administered orally to treat HAE attacks on demand, i.e. at the first signs before the HAE attack fully develops. All currently approved on-demand treatment options require either intravenous or subcutaneous administration.
LiteratureThis section has been translated automatically.
- Cohn DM et al. (2025) CRISPR-Based Therapy for Hereditary Angioedema. N Engl J Med 392:458-467.
- Longhurst HJ et al. (2024) CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema. N Engl J Med 390:432-441.
- Riedl MA et al. (2024) Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks. N Engl J Med 391:32-43.
