Updates provided on berotralstat, an HAE medicine currently being prescribed

A series of updates on berotralstat (brand name: Orladeyo), which is currently being used by people with HAE in many countries around the world, were provided by the pharmaceutical company BioCryst. The key updates shared were:

• New real-world data from over 350 patients with HAE with normal C1 inhibitor showed substantial reductions in attack rates with berotralstat, reinforcing its value for a historically underserved patient segment and providing strong evidence to close both treatment and reimbursement gaps.
• The Prescription Drug User Fee Act (PDUFA) goal date for the company’s new drug application for berotralstat granules in children with HAE aged 2 to 11 is 12 December 2025. Berotralstat would be the first targeted oral prophylactic therapy for children with HAE.

Jon Stonehouse, Chief Executive Officer of BioCryst, said: “In the fifth year since approval, Orladeyo revenue and demand have never been stronger, and this is driven by outstanding execution and increasing confidence in the product.”

(Source: BioCryst)