Sebetralstat for treating hereditary angioedema

Sebetralstat is currently in clinical development as an on-demand treatment for hereditary angioedema (HAE) type I and type II. HAE is a rare genetic condition that usually develops in childhood or early adulthood. It is caused by a defect in a gene that regulates the blood protein C1-inhibitor. Lack of C1-inhibitor leads to the disruption of a complex cascade of events that regulate inflammation. An enzyme (protein) called plasma kallikrein becomes overactive which can ultimately lead to episodic attacks of swelling (oedema) in the hands, feet, gastrointestinal tract, face, and airway. This swelling can cause severe pain and can be potentially life-threatening. This can have a substantial impact on quality of life, particularly because attacks are often difficult to predict. Most current treatments are injected and are focused on long term treatment to reduce the occurrence of an acute attack.