FDA
Oral pellet formulation of Orladeyo OK’d for hereditary angioedema in children
On December 12, 2025, FDA approved an oral pellet formulation of once-daily Orladeyo (berotralstat), a plasma kallikrein inhibitor, for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.
Orladeyo had previously been available only in capsule form and approved for use in patients ≥12 years of age.
Efficacy and safety
Approval was based on data from the APeX-P trial (NCT05453968), the primary objective of which was to describe the PK parameters of berotralstat; secondary objectives were to assess the safety and tolerability of berotralstat and to summarize the efficacy of berotralstat in pediatric patients with HAE. Interim results showed Orladeyo was well tolerated, demonstrated a consistent safety profile across this age group, and resulted in early and sustained reductions in monthly attack rates with no new safety signals identified beyond those previously described in prior adult and adolescent trials. The most commonly reported treatment-emergent adverse event was nasopharyngitis.
Sources:
BioCryst announces FDA approval of Orladeyo® (berotralstat) oral pellets, first and only oral prophylactic treatment for patients with HAE aged 2 to <12 years. [News release]. 2025. https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-fda-approval-orladeyor-berotralstat-oral
Orladeyo (berotralstat) [package insert]. BioCryst. https://www.biocryst.com/wp-content/uploads/sites/13/2025/12/ORLADEYO_USPI.pdf Revised December 2025. Accessed December 17, 2025.