FDA expands berotralstat approval with new pellet formulation for HAE patients of all ages

The FDA has approved berotralstat (Orladeyo) oral pellets as a prophylactic treatment for hereditary angioedema (HAE) in patients aged 12 and older, expanding the drug's reach across all age groups. The pellet formulation was already approved for children aged 2 to under 12, meaning Orladeyo now covers patients from age 2 and up in two oral forms: pellets and capsules (the latter approved since December 2020).

The pellets are designed with accessibility in mind — sprinkle-sized, they can be swallowed directly with water or milk, or mixed into soft, non-acidic food, making them ideal for patients who have difficulty swallowing traditional capsules. The FDA's approval for adolescents and adults was based on chemistry, manufacturing, and safety comparisons between the pellet and capsule formulations.

By the Numbers:

• 0.169 attacks/month with berotralstat vs. 0.691 attacks/month during standard of care (48-week APeX-P study in pediatric patients)
• HAE attacks requiring professional care dropped from 22 (standard of care) to 0 between weeks 37–48 of berotralstat treatment
• 29 pediatric patients (ages 2–<12) enrolled in the APeX-P open-label study

Why it matters: HAE attacks can be life-threatening, and long-term prophylaxis is critical. Expanding berotralstat's formulation options gives clinicians and patients more flexibility, particularly for those who struggle with pill swallowing — a common barrier to adherence across age groups.