BioCryst Pharmaceuticals, Inc. announced today new real-world data showing that patients with hereditary angioedema (HAE) who have normal levels and function of C1 inhibitor (HAE-nC1-INH) had a reduction in monthly attack rates after starting oral, once-daily berotralstat. In addition, new results from the largest body of evidence documenting adverse health outcomes associated with the prophylactic use of attenuated androgens in HAE were presented. These data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) conference in Valencia, Spain.
Efficacy and safety of berotralstat in patients with hereditary angioedema with normal C1 inhibitor: A European case series
Six patients with HAE-nC1-INH were included in the analysis. All had received prior 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 in HAE attack rate, and one patient, who received a concomitant dose of tranexamic acid, showed a 29 percent reduction in HAE attack rate.
No berotralstat-related adverse events occurred in five of the six patients. One patient experienced gastrointestinal symptoms at baseline, which became milder after the first two weeks and did not lead to treatment discontinuation.
Dr. Matthew Buckland, consultant immunologist at the Royal London Hospital, Barts Health NHS Trust, commented that, “Angioedema or spontaneous swelling disorders have a significant impact on quality of life and in the most severe cases can be life-limiting. The diagnosis of HAE-nC1-INH is difficult because there is no “rapid” test and there is often a long delay in receiving appropriate treatment. This real-world evidence shows that patients’ lives can be significantly improved with berotralstat reducing the anxiety caused by the unpredictable nature of their disorder.” Dr. Buckland was the lead contributor to the case series presented at EAACI.
HAE-nC1-INH is the least common subgroup of HAE, where C1 inhibitor levels and function are not impaired. In some cases, no known genetic mutation that causes HAE has been identified. HAE-nC1-INH is four times less common than C1 inhibitor-deficient HAE.
Spam h.Health outcomes and patient and physician perspectives of impaired androgen use in hereditary angioedema
In addition, new results were presented at EAACI demonstrating that adverse health outcomes are associated with impaired use of androgens as a prophylactic for HAE. The study evaluated 108 prospective and retrospective studies published between January 1980 and July 2023 that reported quantitative outcomes associated with impaired androgen use in patients with YAE. These included four clinical trials, 43 observational studies, 37 case reports/series and 24 reviews. Studies on patients’ and physicians’ attitudes and risk perception of attenuated androgens were also included.
This study presents the most recent and largest body of evidence documenting that prophylactic HAE therapy with attenuated androgens is associated with short-term adverse outcomes and serious long-term risks including increased cardiovascular events, liver damage, and cancer. The prevalent and wide-ranging adverse effects associated with impaired androgen use in HAE reinforce that safer and more tolerable treatment options should be preferred and made accessible for HAE prophylaxis.”
Marcus Maurer, Professor of Dermatology and Allergology at Charité – Universitätsmedizin Berlin and Fraunhofer Institute for Translational Medicine and Pharmacology
The study highlights the basis for current World Allergy Agency/EAACI guidelines recommending the use of targeted therapies for long-term first-line prophylaxis and the use of androgens only as long-term second-line prophylaxis.
