BioCryst Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for the use of oral, once-daily ORLADEYO (berotralstat) in pediatric patients with hereditary angioedema (HAE) aged 2 to 11 years. The FDA also granted Priority Review of the application, with a Prescription Drug User Fee Act (PDUFA) target action date of September 12, 2025.
“We are excited to take another step closer to bringing ORLADEYO to younger pediatric patients with HAE. We consistently hear from patients, caregivers and physicians about their desire for a more convenient therapeutic option to treat young children with HAE, and we now may have the opportunity to bring this to them later this year,” said Jon Stonehouse, president and chief executive officer of BioCryst.
The NDA was based on positive interim data from the APeX-P clinical trial, the largest trial evaluating a prophylactic therapy for HAE in patients 2 to 11 years of age. Interim results from APeX-P that were presented at the 2025 American Academy of Allergy, Asthma & Immunology / World Allergy Organization Joint Congress earlier this year showed ORLADEYO was well tolerated and demonstrated a very consistent safety profile across this age group, and resulted in early and sustained reductions in monthly attack rates.
“As detailed in the results from APeX-P, we observed that participants experienced serious HAE attacks at a very early age, with a median age of HAE symptom onset of two years, which suggests there is a larger burden of disease at an earlier age than has been appreciated thus far. If approved, we believe this oral granule formulation of ORLADEYO could help children with HAE and their families better manage their condition and avoid the traumatic experience of acute attacks with emergency care or hospital stays,” said Dr. Helen Thackray, chief research and development officer of BioCryst.
ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE under the age of 12, if approved.
BioCryst has also filed its line extension application for the use of ORLADEYO oral granules in patients with HAE aged 2 to 11 years with the European Medicines Agency. Additional regulatory filings are planned in other global territories, including Japan and Canada.
ORLADEYO received FDA approval in December 2020 for prophylaxis to prevent HAE attacks in adult and pediatric patients 12 years and older and is now commercially available in more than 30 countries.