Orladeyo Pellets Receive FDA Approval for Young HAE Patients—A Milestone for BioCryst

BioCryst Pharmaceuticals has achieved a significant regulatory milestone with FDA clearance of Orladeyo oral pellets designed for prophylactic treatment of hereditary angioedema in children ages 2 through 11. This expansion builds upon the company’s earlier approval for patients aged 12 and above, which was granted in December 2020.

The Clinical Significance

Hereditary angioedema represents a challenging therapeutic landscape for pediatric medicine. This uncommon genetic disorder frequently manifests early in life, with approximately 40% of patients experiencing their initial attack before reaching age 5. The condition’s unpredictable nature, combined with its potential severity and life-threatening complications, creates substantial burden for affected children and their caregivers.

Orladeyo, known generically as berotralstat, functions as a once-daily oral plasma kallikrein inhibitor engineered to prevent HAE attacks rather than merely treat acute episodes. The newly approved pellet formulation specifically addresses the administration challenges inherent to pediatric populations, offering improved tolerability for young patients.

Supporting Evidence from Clinical Trial Data

The FDA’s decision rested on interim findings from the APeX-P study, the most comprehensive investigation to date examining long-term prophylactic therapy in pediatric hereditary angioedema patients. Trial results indicated that Orladeyo maintained a favorable safety profile while consistently reducing monthly attack frequency. Nasopharyngitis emerged as the most frequently reported adverse effect, though the overall tolerability profile remained encouraging.

Global Market Expansion and Financial Performance

The hereditary angioedema treatment market has demonstrated strong commercial momentum for BioCryst’s product. During the third quarter of 2025, Orladeyo generated $159.1 million in net revenue, reflecting robust 37% year-over-year expansion. North American operations accounted for approximately 89% of quarterly global revenue.

Responding to market performance, BioCryst elevated its full-year 2025 revenue projection for Orladeyo to a range between $590 million and $600 million. The product has secured regulatory presence in more than 45 countries globally.

Regulatory Momentum Continues

Beyond FDA approval, BioCryst has submitted regulatory applications to the European Medicines Agency and Japan’s PMDA for the oral pellet formulation. Additional market submissions are anticipated in Canada and other jurisdictions, positioning hereditary angioedema treatment options for broader international availability.

Stock Performance Context

BCRX shares closed the previous trading session at $7.58, down 1.04% for the day. Over the preceding 12-month period, the stock traded within a range of $6.00 to $11.31.