BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to

Core Insights - BioCryst Pharmaceuticals has received FDA approval for ORLADEYO® (berotralstat), the first and only targeted oral prophylactic therapy for pediatric patients with hereditary angioedema (HAE) aged 2 to less than 12 years [1][4][12] Group 1: Product Details - ORLADEYO is now available in an oral pellet formulation, designed for easier administration to children, allowing it to be swallowed directly or sprinkled over soft food [6][9] - The approval was based on positive interim data from the APeX-P clinical trial, which demonstrated that ORLADEYO was well tolerated and resulted in early and sustained reductions in monthly attack rates for pediatric patients [5][7] Group 2: Market Context - Approximately 40% of children with HAE experience their first attack by age 5, highlighting the need for effective treatment options that are less burdensome than intravenous or subcutaneous therapies [2] - The introduction of ORLADEYO oral pellets is expected to change the management of HAE in younger patients, providing a more convenient option for families and caregivers [9] Group 3: Company Commitment - BioCryst is committed to supporting patients through its Empower Patient Services, which offers comprehensive assistance for navigating insurance and treatment needs [10] - The company has plans for additional regulatory filings for ORLADEYO in other regions, including Europe and Japan, indicating a strategy for global market expansion [8]