BioCryst Pharmaceuticals(BCRX) Newsfilter·2025-02-10 13:30Core Insights - BioCryst Pharmaceuticals, Inc. announced the presentation of five abstracts on ORLADEYO® (berotralstat) for hereditary angioedema (HAE) at the 2025 AAAAI/WAO joint congress [1][2] - A late-breaking abstract will present results from the APeX-P trial focusing on pediatric patients aged 2 to <12 years [2] - The congress is scheduled for February 28 to March 3, 2025, in San Diego [2] Company Overview - BioCryst Pharmaceuticals is a biotechnology company dedicated to improving the lives of individuals with hereditary angioedema and other rare diseases [11] - The company specializes in structure-guided drug design to develop innovative small-molecule and protein therapeutics [11] - ORLADEYO® is the first oral, once-daily plasma kallikrein inhibitor approved for the prophylactic treatment of HAE in patients aged 12 years and older [3][4] Product Information - ORLADEYO® (berotralstat) is indicated for the prevention of HAE attacks in adults and pediatric patients aged 12 years and older [4] - The drug works by inhibiting plasma kallikrein, thus preventing HAE attacks with a once-daily dosage [3] - The safety and effectiveness of ORLADEYO in pediatric patients under 12 years have not been established [10] Clinical Data Presentation - The five abstracts to be presented include: - HAE attack rates in pediatric patients aged 2 to <12 years [8] - Real-world attack rates before and after berotralstat initiation among patients with C1-inhibitor deficiency [8] - Real-world attack rates for patients without C1-inhibitor deficiency [8] - Patient willingness to change long-term prophylaxis based on disease burden and treatment effectiveness [8] - Patient-reported impacts of berotralstat on attack frequency and severity [8]