FDA Approves BioCryst’s Oral Pellet Form of Orladeyo, First Oral Prophylactic Therapy for Children Aged 2–11 with HAE

Durham, NC — Dec. 12, 2025 — The U.S. Food and Drug Administration (FDA) has approved an oral pellet formulation of Orladeyo (berotralstat) from BioCryst Pharmaceuticals for prophylactic use in children aged 2 to 11 years with hereditary angioedema (HAE). This new formulation marks the first oral preventive therapy approved in the U.S. for this pediatric age group, offering a more convenient and child-friendly alternative to injections or intravenous treatments.

HAE is a rare genetic disorder characterized by unpredictable and potentially life-threatening swelling episodes affecting the face, throat, extremities, and gastrointestinal tract. Until now, treatment options for children under 12 years old have relied mainly on injectable or infused therapies, which can be burdensome for both patients and caregivers. The newly approved oral pellets can be mixed with soft food or swallowed with water or milk, significantly improving administration ease for young children.

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Orladeyo was originally approved in December 2020 as a once-daily capsule for patients aged 12 years and older. The pediatric pellet formulation was approved based on data from the APeX-P clinical trial, which demonstrated sustained reductions in monthly HAE attack rates with a favorable safety profile. BioCryst has also filed applications seeking similar pediatric approvals in Europe and Japan.

Following the FDA decision, BioCryst’s shares rose approximately 5% in pre-market trading, reflecting investor optimism about the company’s expanded pediatric market potential. Industry analysts believe this approval will further strengthen Orladeyo’s leadership position in the HAE therapeutic space and support broader global adoption of the pediatric oral formulation.

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Industry Insight — Hillgene Perspective

The FDA’s approval of an oral pellet form of Orladeyo for young children highlights a broader impetus toward pediatric-friendly formulations in rare disease care. For contract manufacturing and development organizations (CDMOs), this trend elevates the importance of advanced oral formulation technologies — including taste-optimized pellets, dispersion in food matrices, and stringent pediatric quality standards. Hillgene believes that companies capable of delivering robust pediatric formulation and manufacturing solutions will gain strategic advantages as regulatory frameworks increasingly emphasize ease of use and treatment adherence for young populations.

Source: Reuters, FDA approves BioCryst’s oral pellet version of rare disease drug for children, December 12, 2025.