Orladeyo's Pediatric Win Signals BioCryst's Growth Trajectory in HAE Market

BioCryst Pharmaceuticals’ latest FDA approval marks a significant expansion in its hereditary angioedema portfolio. The company’s oral pellet formulation of Orladeyo has been greenlit for prophylactic treatment in children aged 2 to under 12—a population that previously lacked age-appropriate options. This approval addresses a critical gap, as roughly 40% of HAE patients experience their first attack before turning 5.

Revenue Momentum Reflects Market Confidence

Financial performance underscores investor optimism. In Q3 2025, Orladeyo generated $159.1 million in net revenue, representing 37% year-over-year growth. The U.S. market accounted for 89% of global sales, while BioCryst elevated its full-year 2025 revenue guidance to $590-600 million. BCRX shares traded in the $6.00-$11.31 range over the past 12 months, closing yesterday’s session at $7.58, down 1.04%.

Clinical Foundation Behind the Pediatric Push

The pediatric approval rests on solid clinical evidence. The APeX-P trial, the most comprehensive long-term prophylactic study for pediatric HAE to date, demonstrated that berotralstat—Orladeyo’s active ingredient—delivered consistent safety and early, sustained reductions in monthly attack rates. Nasopharyngitis emerged as the most frequently reported treatment-related adverse event. As a once-daily oral plasma kallikrein inhibitor, the drug offers convenience compared to traditional intervention methods.

Why the Pellet Formulation Matters

HAE is unpredictable and potentially life-threatening, creating substantial burdens for young patients and families. The new pellet form addresses a practical challenge: pediatric-friendly administration. Unlike capsules or tablets, pellets can be mixed with food, improving compliance in this vulnerable population.

Global Expansion Strategy Taking Shape

This FDA clearance builds on Orladeyo’s December 2020 approval for patients aged 12 and older, now distributed across 45+ countries. BioCryst has submitted regulatory applications to Europe’s EMA and Japan’s PMDA, with filings planned for Canada and additional territories. This multi-market approach signals confidence in the product’s long-term commercial potential across diverse healthcare systems.