BioCryst Pharmaceuticals has announced new data showing consistently high attack-free rates among patients with hereditary angioedema (HAE) on long-term prophylaxis with berotralstat (Orladeyo®).

The data, announced via news release, demonstrated sustained reductions in attack rates over a 96-week treatment period and will be presented at the 13th C1-inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary, this May.

“These data demonstrate how our oral, once-daily prophylactic treatment can consistently help patients spend more of their days without disruption from HAE attacks, regardless of their age, gender and experience with prior prophylactic treatment, including among pediatric patients aged 12-17,” said Dr. Ryan Arnold, chief medical officer of BioCryst. “These additional analyses of long-term data reflect our continued commitment to define the potential benefits and bring Orladeyo to as many HAE patients around the world as possible.”

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HAE is a rare disease caused by mutations in the SERPING1 gene, which codes for C1-INH, an enzyme that prevents inflammation. The condition is characterized by repeated episodes of swelling in the airway, skin, and gastrointestinal tract that last 2 to 5 days.

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The data from BioCryst are based on analyses of the APeX-S clinical trial, a phase 2, open-label study on 287 individuals with HAE type I or II receiving 150 mg of berotralstat daily for up to 96 weeks in the US and up to 240 weeks in all other countries.

The primary outcome measure of the study was attack-free status among patients stratified by age, gender, and previous prophylaxis. Attack-free status was consistently high among all patient subgroups assessed in the study.

Berotralstat is a daily oral therapy designed to prevent HAE attacks in patients 12 years and older. It works by reducing the activity of kallikrein in plasma, thereby reducing inflammation and the risk of attacks.


BioCryst presents new long-term data demonstrating consistently high attack-free status among hereditary angioedema patients with ORLADEYO® (berotralstat). News release. BioCryst Pharmaceuticals, Inc.; April 27, 2023.