FDA Approves ORLADEYO Oral Pellets for Adults and Adolescents: New Formulation Option for Hereditary Angioedema Prevention
BioCryst Pharmaceuticals, based in Research Triangle Park, North Carolina, announced on October 21 that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application for the 150 mg oral pellet formulation of once-daily ORLADEYO (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
The new pellet formulation gives patients who have difficulty swallowing, prefer not to take a capsule, or wish to remain on pellets beyond age 12 another once-daily oral option. The pellets are sprinkle-like in size and can be poured directly into the mouth and swallowed with water or milk, or sprinkled over a spoonful of soft, non-acidic food. The approval was based on chemistry, manufacturing and controls information and data on the safety and tolerability of the pellets compared with the capsules.
ORLADEYO is the first and only targeted oral prophylactic therapy for HAE patients aged 2 and above, working by decreasing plasma kallikrein activity. Its capsule form was first approved by the FDA in December 2020 for patients 12 and older and is now approved in more than 45 countries; the pellet form was approved in December 2025 for children aged 2 to under 12. Across two clinical studies cited by the company, 150 mg capsules produced a mean attack-rate reduction of 91% at 96 weeks and an 88% reduction in rescue medication use, with gastrointestinal reactions and back pain the most frequently reported adverse reactions.
BioCryst President and Chief Executive Officer Charlie Gayer said the approval strengthens the company's ability to offer personalized treatment choice with the proven efficacy of ORLADEYO in a formulation that is easier for patients who have difficulty swallowing or prefer not to take a capsule. The company noted ORLADEYO is not established for treating acute HAE attacks, and doses above 150 mg once daily are not recommended because of the potential for QTc interval prolongation.
Published by GSM · USA (original Chinese edition)
Photo: 图：处方药胶囊（资料图）。美国国家过敏与传染病研究所 NIAID 摄，CC BY 2.0，取自维基共享资源。Photo: Prescription capsules (file photo). NIAID, CC BY 2.0, via Wikimedia Commons.
