FDA accepts BioCryst’s application to expand ORLADEYO to young HAE patients

The U.S. Food and Drug Administration (FDA) has accepted Durham-based BioCryst Pharmaceuticals’ application to expand the use of ORLADEYO (berotralstat) to treat children ages 2 to 11 with a rare genetic condition known as hereditary angioedema (HAE). The FDA also granted the application Priority Review with a Prescription Drug User Fee Act date of September 12, 2025.

HAE causes sudden and severe swelling in various parts of the body, including the face, hands, stomach and airways, which can be painful and sometimes life-threatening. If approved, ORLADEYO, which is taken orally once daily, would be the first targeted oral prophylactic therapy for children with HAE under the age of 12. It is currently approved for use in adults and children aged 12 and older and is commercially available in more than 30 countries.

“We are excited to take another step closer to bringing ORLADEYO to younger pediatric patients with HAE,” said Jon Stonehouse, president and CEO of BioCryst. “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.”

Promising clinical trial results

HAE attacks occur because people with HAE lack or have a problem with a protein called C1 inhibitor, which normally helps control inflammation. Without it, fluid can leak into tissues and cause swelling. ORLADEYO prevents HAE attacks by targeting a key part of the process that leads to swelling.

The New Drug Application was based on positive interim data from the APeX-P clinical trial, which showed that the ORLADEYO oral granule formulation developed for children under 12 was well tolerated and demonstrated a very consistent safety profile in children under the age of 12, with a consistent safety profile and early, sustained reductions in monthly attack rates.

“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,” said Helen Thackray, M.D., chief research and development officer of BioCryst. “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.”

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.

BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of people living with hereditary angioedema and other rare diseases. It was founded in 1986 and moved its headquarters to Durham in 2010. The company discovers and develops small-molecule medicines that treat rare diseases.