FDA Accepts NDA for ORLADEYO® Oral Granules in Young Hereditary Angioedema Patients

BioCryst Pharmaceuticals, Inc., a biotechnology company in Research Triangle Park, North Carolina, has announced a significant advancement in its efforts to provide treatment for pediatric patients suffering from hereditary angioedema (HAE). The U.S. Food and Drug Administration (FDA) has accepted BioCryst's New Drug Application (NDA) for ORLADEYO® (berotralstat), a once-daily oral medication aimed at children aged 2 to 11 years with HAE. This acceptance comes alongside the FDA granting a Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of September 12, 2025.

Jon Stonehouse, BioCryst’s president and chief executive officer, expressed enthusiasm for this development, emphasizing the potential of ORLADEYO to offer a more convenient treatment option for younger patients with HAE. The feedback from patients, caregivers, and physicians has highlighted a demand for such a therapeutic solution, and the company hopes to meet this need by the end of the year.

The NDA submission is supported by positive interim data from the APeX-P clinical trial, recognized as the largest study focused on a prophylactic therapy for HAE in the stated age group. Findings from APeX-P were presented earlier this year at a major allergy congress, demonstrating that ORLADEYO is well tolerated among young patients, offering a consistent safety profile and contributing to sustained reductions in monthly attack rates.

Dr. Helen Thackray, BioCryst's chief research and development officer, noted the trial's observation that serious HAE attacks begin at a very young age, with symptoms often appearing as early as two years old. This suggests a greater burden of the disease at an early age than previously acknowledged. She believes that the approval of ORLADEYO's oral granule formulation could significantly aid children with HAE and their families in managing the condition, potentially reducing the need for emergency interventions or hospitalizations due to acute attacks.

If approved, ORLADEYO would become the first targeted oral prophylactic treatment available for children under 12 years with HAE. BioCryst is also pursuing regulatory approval in Europe, having submitted a similar application to the European Medicines Agency. Further filings are planned for other regions, including Japan and Canada.

ORLADEYO has already been authorized by the FDA in December 2020 for use in preventing HAE attacks among adult and pediatric patients aged 12 years and older. The medication is currently available in over 30 countries, illustrating BioCryst's commitment to expanding access to effective treatments for rare diseases.

BioCryst Pharmaceuticals is dedicated to improving the lives of individuals dealing with rare conditions such as hereditary angioedema. Through innovative drug design, BioCryst aims to develop pioneering small-molecule and protein therapies that address complex medical challenges. The prospect of ORLADEYO's approval for younger patients marks another step forward in BioCryst's mission to deliver groundbreaking healthcare solutions.