BioCryst Announces FDA Acceptance of NDA for ORLADEYO® (berotralstat) Oral Granules in Patients with Hereditary Angioedema Aged 2 to 11 Years

Core Viewpoint - The FDA has accepted BioCryst Pharmaceuticals' New Drug Application for ORLADEYO, which, if approved, would be the first targeted oral prophylactic therapy for pediatric patients with hereditary angioedema (HAE) under the age of 12, with a PDUFA target action date of September 12, 2025 [1][5]. Group 1: FDA Approval and Application - The FDA granted Priority Review for the NDA of ORLADEYO for pediatric patients aged 2 to 11 years [1]. - ORLADEYO is already approved for patients aged 12 years and older and is commercially available in over 30 countries [6]. Group 2: Clinical Trial Data - The NDA is based on positive interim data from the APeX-P clinical trial, which is the largest trial evaluating prophylactic therapy for HAE in the specified age group [3]. - Interim results indicated that ORLADEYO was well tolerated, showing a consistent safety profile and early, sustained reductions in monthly attack rates [3]. Group 3: Market Potential and Company Strategy - The company aims to provide a more convenient therapeutic option for young children with HAE, responding to demand from patients, caregivers, and physicians [2]. - BioCryst has also filed for a line extension application with the European Medicines Agency and plans additional regulatory filings in Japan and Canada [5].