FDA accepts NDA for ORLADEYO granules in HAE ages 2-11; priority review, PDUFA Sep 12, 2025

BIOCRYST PHARMACEUTICALS INC

FDA accepted NDA for once-daily ORLADEYO oral granules for HAE patients aged 2-11; priority review granted with PDUFA target Sep 12, 2025.
If approved, ORLADEYO would be first targeted oral prophylactic therapy for HAE under age 12.
NDA based on positive interim APeX-P trial data showing well-tolerated, consistent safety, and reduced monthly attack rates.
BioCryst also filed line extension with EMA; regulatory filings planned in Japan and Canada.
ORLADEYO already approved in US for patients 12+ and commercially available in over 30 countries.

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