Abstract
Hereditary angioedema (HAE) is rare disorder caused by a SERPING1 gene mutation that triggers severe swelling of the skin and upper airway. Treatment options for HAE with deficient and dysfunctional C1-inhibitor are expanding to include small-molecule drugs that inhibit protein interactions in the kallikrein-kinin system. Discovered by BioCryst Pharmaceuticals, BCX7353 is a synthetic, once-daily, small molecule drug that can be taken as an oral capsule to treat HAE attacks and for prophylaxis. This article will summarize recent and current BCX7353 clinical trials. Overall, results indicate BCX7353 is a promising form of therapy with a rapid 1 h onset of action, long duration of action, and acceptable tolerance.
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