FDA Approves BioCryst's ORLADEYO Oral Pellets for Pediatric HAE Prevention

Reuters
2025/12/12
FDA Approves BioCryst's ORLADEYO Oral Pellets for Pediatric HAE Prevention

BioCryst Pharmaceuticals Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for an oral pellet formulation of ORLADEYO® (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema $(HAE)$ aged 2 to less than 12 years. This approval makes ORLADEYO the first and only targeted oral prophylactic therapy available for patients with HAE aged 2 and older. The new oral pellet formulation is designed to provide a child-friendly method of administration and demonstrated early and sustained reductions in monthly attack rates in clinical studies. BioCryst has also filed applications for the use of ORLADEYO oral pellets in children with HAE in Europe and Japan, with additional regulatory filings planned in other global territories, including Canada.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioCryst Pharmaceuticals Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9601244-en) on December 12, 2025, and is solely responsible for the information contained therein.

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