FDA Approves BioCryst's NDA For Use Of Oral Pellet Formulation Of Once-Daily ORLADEYO For Prophylactic Therapy In Pediatric Patients With Hereditary Angioedema Aged 2 To <12 Years

–ORLADEYO now first and only targeted oral prophylactic therapy for patients with HAE aged 2 and older–

-Oral pellet formulation provides child-friendly method of administration-

–Showed early and sustained reductions in monthly attack rates in APeX-P–

RESEARCH TRIANGLE PARK, N.C., Dec. 12, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for the use of an oral pellet formulation of once-daily ORLADEYO® (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.

HAE often presents during childhood, with approximately 40% of children with HAE having their first attack by age 5. HAE can significantly impair normal daily living due to the physical manifestations and psychosocial impact resulting from the disease's unpredictable, debilitating, and potentially life-threatening nature. Until now, the only targeted treatment options for those under age 12 were administered intravenously or through subcutaneous injection, which can be burdensome for younger patients living with HAE and their caregivers.