BioCryst FDA Accepts NDA for ORLADEYO for Hereditary Angioedema Aged 2 to 11

The FDA has accepted BioCryst Pharmaceuticals' (BCRX) New Drug Application for ORLADEYO® oral granules to treat hereditary angioedema (HAE) in children aged 2-11 years. The FDA granted Priority Review with a PDUFA target date of September 12, 2025.

The application is based on positive interim data from the APeX-P clinical trial, showing ORLADEYO was well-tolerated with sustained reductions in monthly attack rates. The trial revealed HAE symptoms typically onset at age two, indicating a significant early disease burden. If approved, ORLADEYO would become the first targeted oral prophylactic therapy for HAE patients under 12 years old.

BioCryst has also filed for approval in Europe, with plans for additional submissions in Japan and Canada. ORLADEYO is currently approved for patients 12 years and older in over 30 countries since December 2020.

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