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Berotralstat is an oral small molecule inhibitor of plasma kallikrein, developed for the prophylaxis (prevention) of attacks in patients with hereditary angioedema (HAE), a rare genetic disorder characterized by recurrent episodes of severe swelling. By binding to and inhibiting plasma kallikrein, berotralstat reduces the production of bradykinin—a potent vasodilator responsible for increased vascular permeability and swelling in HAE. Berotralstat is indicated for use in adults and pediatric patients aged 12 years and older to prevent HAE attacks. It is not used for acute treatment of attacks. The drug was developed by BioCryst Pharmaceuticals and is marketed under the brand name Orladeyo.
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