Berotralstat: A promising oral treatment for hereditary angioedema with normal C1-inhibitor

Jun 8 2024BioCryst Pharmaceuticals BioCryst Pharmaceuticals, Inc. today announced new real-world evidence showing that patients with hereditary angioedema who have normal C1-inhibitor level and function had a reduction in monthly attack rates after starting oral, once-daily berotralstat. Additionally, new results were presented from the largest body of evidence documenting adverse health outcomes associated with prophylactic use of attenuated androgens in HAE.

No adverse events related to berotralstat were noted in five of the six patients. One patient experienced gastrointestinal symptoms upon initiation, which became milder after the first two weeks and did not lead to treatment discontinuation. HAE-nC1-INH is the least common subgroup of HAE, where the levels of and function of C1-inhibitor are not impeded. In some cases, no known genetic mutation causing HAE has been identified. HAE-nC1-INH is four times less common that C1-inhibitor deficient HAE.

This study presents the latest and largest body of evidence documenting that HAE prophylactic treatment with attenuated androgens is associated with short-term adverse outcomes and serious long-term risks that include increased cardiovascular events, liver damage, and cancer. The prevalent and wide-ranging adverse outcomes associated with attenuated androgen use in HAE reinforce that safer and more tolerable treatment options should be preferred and made accessible for HAE prophylaxis.

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