Core Viewpoint - BioCryst Pharmaceuticals has received FDA approval for ORLADEYO, the first and only targeted oral prophylactic therapy for pediatric patients aged 2 to less than 12 years with hereditary angioedema (HAE) [1][4]. Group 1: Product Information - ORLADEYO is an oral pellet formulation designed for once-daily administration, providing a child-friendly method of treatment [1][6]. - The approval is based on positive interim data from the APeX-P clinical trial, which demonstrated early and sustained reductions in monthly attack rates for pediatric patients [5][7]. - ORLADEYO has been prescribed to over 3,500 patients in the U.S. since its initial launch for older patients [5]. Group 2: Market Context - Approximately 40% of children with HAE experience their first attack by age 5, highlighting the need for effective treatment options for younger patients [2]. - Prior to this approval, treatment options for children under 12 were limited to intravenous or subcutaneous methods, which can be burdensome [2][9]. - The new oral pellet formulation aims to alleviate the treatment burden and improve the quality of life for children with HAE and their caregivers [7][9]. Group 3: Regulatory and Future Plans - BioCryst has filed applications for ORLADEYO oral pellets with the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency, with additional regulatory filings planned in other territories, including Canada [7][8]. - The company is committed to providing comprehensive support through its Empower Patient Services program, which assists patients and healthcare providers with various needs related to ORLADEYO [10].