BioCryst Pharmaceuticals Receives FDA Approval for ORLADEYO 150 mg Pellets for HAE

RESEARCH TRIANGLE PARK, N.C., Oct. 21, 2026 /CourierPR/ -- BioCryst Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for the 150 mg oral pellet formulation of ORLADEYO® for prophylactic therapy in adults and adolescents aged 12 years and older. This approval expands the company's portfolio of targeted oral prophylactic therapy options, providing an alternative administration option for patients who may have difficulty swallowing, prefer not to take a capsule, or choose to remain on a pellet formulation beyond age 12.

ORLADEYO® (berotralstat) is the first and only targeted oral prophylactic therapy for patients with hereditary angioedema (HAE) aged 2 and above. The capsule formulation of ORLADEYO received FDA approval for prophylaxis to prevent HAE attacks in adult and pediatric patients 12 years and older in December 2020 and is now approved in more than 45 countries around the world. In December 2025, the oral pellet formulation of ORLADEYO was approved by the FDA for prophylactic therapy in pediatric patients in the U.S. with HAE aged 2 to <12 years.

The new 150 mg ORLADEYO pellet formulation provides an alternative administration option for adults and adolescents who may have difficulty swallowing, prefer not to take a capsule, or choose to remain on a pellet formulation beyond age 12. The pellets are sprinkle-like in appearance and size, allowing patients to pour them directly into the mouth and swallow immediately with water or milk, or sprinkle them over a spoonful of soft, non-acidic food.

Across two clinical studies, ORLADEYO capsules (150 mg) resulted in sustained attack prevention in patients 12 years and older with a mean attack-rate reduction of 91% at 96 weeks and an 88% reduction in rescue medication use. ORLADEYO was safe and well tolerated in both trials. The most frequently reported adverse reactions in patients receiving ORLADEYO compared with placebo were gastrointestinal reactions and back pain. These gastrointestinal reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time, and typically self-resolved.

BioCryst is committed to providing support every step of the way and offers Empower Patient Services, a single stop for all patient and healthcare provider (HCP) ORLADEYO needs. It combines a specialty pharmacy and patient support program with a dedicated team of experts who provide individualized and streamlined support navigating the insurance process, affordability, adherence, and related needs.

The approval of the 150 mg ORLADEYO pellet formulation is a significant milestone for BioCryst, as it expands the company's portfolio of targeted oral prophylactic therapy options for HAE patients. This new formulation offers an alternative administration option for patients who may have difficulty swallowing or prefer not to take a capsule, thereby enhancing the treatment options available to HAE patients.