ORLADEYO
ORLADEYO (berotralstat) is a once-daily oral therapy developed by BioCryst for the prevention of hereditary angioedema (HAE) attacks. HAE is a rare genetic condition in which C1 inhibitor deficiency leads to uncontrolled plasma kallikrein activation and excess bradykinin production, triggering sudden severe swelling in the skin, gut, and airway — laryngeal attacks can be immediately fatal. Berotralstat directly inhibits plasma kallikrein to prevent attacks before they start; it is the only once-daily oral HAE prophylactic approved in the United States, and BioCryst is pursuing label extension to children aged 2 to 11.
Upcoming catalysts
Programs
Hereditary Angioedema
Hereditary angioedema attacks cause severe swelling in the skin, gut, and airway driven by uncontrolled bradykinin from C1 inhibitor deficiency; laryngeal attacks are potentially fatal and prior subcutaneous or IV prophylaxis required injections every 1–4 weeks. ORLADEYO is the only once-daily oral HAE prophylactic approved in the US for adults and adolescents aged 12+; the pediatric sNDA filing in 2026 supported by Phase 3 APeX-P data targets label expansion to children aged 2 to 11.