New data on efficacy and safety of HAE medicine berotralstat presented at key European congress

The long-term efficacy and safety of berotralstat (Orladeyo) was examined in a series of new studies, presented at the 2025 European Academy of Allergy and Clinical Immunology (EAACI) Congress.

Four studies were presented by the pharmaceutical company BioCryst:

• Berotralstat Use Reduced Number of HAE Attacks Requiring Treatment or Professional Care in Pediatric Patients: Interim Results from APeX-P
• Assessment of the Effectiveness and Tolerability of Berotralstat for Long-term Prophylaxis in Hereditary Angioedema: Findings from the Berolife Study
  • Impact of Berotralstat on Quality of Life in Patients with Hereditary Angioedema
  • Patients With HAE Report Positive Perceptions Following Berotralstat Treatment: Results from a Focus Group

Helen Thackray, Chief Research and Development Officer of BioCryst, said: “These data from both clinical trials and real-world settings continue to strengthen the evidence that Orladeyo is an effective and well-tolerated long-term prophylactic treatment for HAE. Importantly, patients report not only fewer and less severe attacks, but also meaningful improvements in daily functioning and emotional well-being. This contributes to the growing body of evidence supporting the use of Orladeyo as a long-term prophylactic option for adolescents and adults living with HAE.”

(Source: BioCryst)