FDA Approves Berotralstat Oral Pellets for Adolescent and Adult HAE
Key Clinical Summary
- The US FDA approved once-daily berotralstat 150 mg oral pellets for hereditary angioedema (HAE) prophylaxis in patients aged 12 years and older.
- The formulation offers an alternative for patients with swallowing difficulties, those who prefer avoiding capsules, or those continuing pellets beyond age 12.
- Approval relied on chemistry, manufacturing, and controls information and comparative safety and tolerability data; reported long-term efficacy findings involved capsules.
BioCryst Pharmaceuticals announced October 21, 2026, that the US Food and Drug Administration approved its supplemental New Drug Application for berotralstat (Orladeyo) 150 mg oral pellets for HAE prophylaxis in adults and adolescents aged 12 years and older. The approval expands administration choices for these patients, who can now receive once-daily berotralstat as either capsules or pellets to prevent HAE attacks.
Patients can use the newly approved pellets by pouring directly into the mouth and swallowing immediately with water or milk, or the pellets can be sprinkled over a spoonful of soft, nonacidic food.
The agency based its decision in part on data demonstrating the pellet formulation’s safety and tolerability compared with berotralstat capsules.
BioCryst also cited long-term findings from 2 clinical studies of berotralstat 150 mg capsules in patients aged 12 years and older. Those studies demonstrated that treatment berotralstat produced a mean reduction in HAE attacks of 91% at 96 weeks and an 88% reduction in rescue medication use.
The company reported that berotralstat was safe and well tolerated in both trials. The most frequently reported adverse reactions among recipients compared with placebo were gastrointestinal reactions and back pain. Gastrointestinal reactions generally occurred early after treatment initiation, became less frequent over time, and typically resolved spontaneously.
Clinical Implications
For US clinicians managing HAE, the approval adds an administration option for patients who have difficulty swallowing capsules or prefer a different formulation. It also allows patients using pellets to continue that formulation beyond age 12.
The FDA initially approved berotralstat capsules in December 2020 for HAE prophylaxis in patients aged 12 years and older. In December 2025, the agency approved oral pellets for US children aged 2 to younger than 12 years.
According to BioCryst, berotralstat is the first and only targeted oral prophylactic therapy with options for patients with HAE aged 2 years and older. The capsule formulation is approved in more than 45 countries.
This latest approval extends berotralstat pellet availability across pediatric, adolescent, and adult age groups in the United States.
Reference
BioCryst announces U.S. FDA approval of ORLADEYO® 150 mg oral pellets for adults and adolescents with HAE. Press release. Published October 21, 2026. https://ir.biocryst.com/node/28361/pdf
