BioCryst reports upcoming key milestones

BioCryst Pharmaceuticals provided a corporate update.

Jon Stonehouse, President and Chief Executive Officer of BioCryst, said: “We ended 2024 with the strongest execution and performance in the company’s history, and this year is off to a fantastic start, with our pediatric label expansion for Orladeyo anticipated later this year.”

The company has received final reimbursement for Orladeyo in Portugal. Orladeyo is now reimbursed in all major countries in Western Europe, except the Netherlands, which is expected in 1H 2025.

Dr Helen Thackray, Chief R&D Officer of BioCryst, said: “There is a tremendous unmet need for an oral option for children with HAE, so we are excited to bring Orladeyo to children as young as two years old.”

The company is on track to submit a new drug application (NDA) in 2025 to the U.S. Food and Drug Administration (FDA) to expand the Orladeyo label to children with HAE aged 2 to 11 using an oral granule formulation. Additional regulatory filings are planned in global territories, including Europe, Japan and Canada. Orladeyo would be the first targeted oral prophylactic therapy for children with HAE.

(Source: BioCryst)