BioCryst Pharmaceuticals, Inc. today announced new real-world evidence showing that patients with hereditary angioedema (HAE) who have normal C1-inhibitor (HAE-nC1-INH) 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.
These data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in Valencia, Spain. Six patients with HAE-nC1-INH were included in the analysis. All had received previous long-term prophylaxis (LTP), and one remained on concurrent LTP.
After six months of treatment with berotralstat, five patients showed a 75 to 100 percent reduction of their HAE attack rate, and one patient, who was on a concurrent dose of tranexamic acid, showed a 29 percent reduction in their HAE attack rate. 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.
Dr. Matthew Buckland, consultant immunologist at Royal London Hospital, Barts Health NHS Trust commented that, “Angioedema or spontaneous swelling disorders have a major impact on quality of life and in the more severe cases can be life limiting. Diagnosis of HAE-nC1-INH is difficult.