BioCryst Pharmaceuticals, Inc. (BCRX) announced that the FDA has approved Orladeyo oral pellets for prophylactic treatment of hereditary angioedema or HAE in pediatric patients aged 2 to under 12 years.
HAE is a rare genetic disorder that often presents in childhood, with about 40% of patients experiencing their first attack by age 5. The disease is unpredictable, debilitating, and potentially life-threatening, significantly impacting daily life for children and their families.
Orladeyo or berotralstat is a once-daily oral plasma kallikrein inhibitor designed to prevent HAE attacks. The new pellet formulation provides a child-friendly administration option.
FDA's approval for New Drug Application was supported by interim data from the APeX-P clinical trial, the largest study to date evaluating long-term prophylactic therapy for pediatric HAE.
The company said Orladeyo was well tolerated, demonstrated a consistent safety profile, and produced early and sustained reductions in monthly attack rates. The most common treatment-emergent adverse event was nasopharyngitis.
This pediatric approval builds on Orladeyo's earlier FDA clearance in December 2020 for patients aged 12 and older, a therapy now available in more than 45 countries worldwide.
Regulatory filings for the oral pellet formulation have also been submitted to the European Medicines Agency and Japan's PMDA, with additional filings planned in Canada and other territories.
Orladeyo generated $159.1 million in net revenue in the third quarter of 2025, an increase of 37% year-over-year.
In the first week of November, BioCryst had raised its full-year 2025 revenue guidance for Orladeyo to between $590 million and $600 million.U.S. sales contributed about 89% of global revenue in the quarter.
BCRX has traded between $6.00 and $11.31 over the past year. The stock closed yesterday's trading at $7.58, down 1.04%.
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