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BioCryst Presents New Real-world Evidence Showing Reductions in Attack Rates in HAE Patients with Normal C1-Inhibitor after Beginning ORLADEYO® (berotralstat) Treatment
Additional results from largest body of evidence on attenuated androgen use in HAE reinforce need for access to safer and more tolerable HAE prophylaxis
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"Additional results from largest body of evidence on attenuated androgen use in HAE reinforce need for access to safer and more tolerable HAE prophylaxis options\nRESEARCH TRIANGLE PARK, N.C., June 02, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) 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 ORLADEYO® (berotralstat). The data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in Valencia, Spain. “The diagnosis of HAE patients with normal C1-inhibitor is complicated and often delayed by the lack of an easily measurable biochemical marker. This multicenter case series provides clinically relevant evidence that berotralstat can also reduce the frequency and duration of episodes in the C1 normal population,” said Dr. Isabelle Boccon-Gibod, department of internal medicine and immunology, Grenoble Alps University Hospital, who presented the findings at EAACI. 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. Adverse Health Outcomes and Patient and Physician Perspectives of Attenuated Androgen Use in Hereditary Angioedema Additional new results presented at EAACI demonstrate the adverse health outcomes associated with attenuated androgen use as a prophylactic treatment for HAE. The study also documents that these adverse outcomes cause increased reluctance among physicians to use attenuated androgens in clinical practice. The study highlights the importance of access to recent targeted HAE prophylactic therapies, in line with current World Allergy Organization/EAACI guidelines which recommend that targeted thera...