FDA Approves Berotralstat Oral Pellets for Adults, Adolescents With HAE

The US Food and Drug Administration (FDA) has approved a 150 mg oral pellet formulation of once-daily berotralstat (Orladeyo) for prophylaxis of hereditary angioedema (HAE) attacks in patients aged ≥ 12 years, BioCryst Pharmaceuticals announced on October 21, 2026.¹

The approval adds a second dosage form for a population previously limited to capsules, which received FDA approval in December 2020.¹ In December 2025, the FDA approved berotralstat pellets for pediatric patients aged 2 to < 12 years, and the new 150 mg strength allows those patients to remain on pellets beyond age 12 years.¹˒² According to BioCryst, berotralstat is the only targeted oral prophylactic therapy for patients with HAE aged ≥ 2 years.¹

“The approval of ORLADEYO pellets for adults and adolescents strengthens our ability to provide personalized treatment choice by offering the proven efficacy of ORLADEYO in a new formulation to make administration easier for patients who have difficulty swallowing or prefer not to take a capsule,” said Charlie Gayer, president and chief executive officer of BioCryst.¹

Basis for the Berotralstat 150 mg Oral Pellet Approval

BioCryst submitted the pellet formulation through a supplemental New Drug Application (sNDA). The FDA based its decision on chemistry, manufacturing, and controls information, along with data comparing the safety and tolerability of the pellets with berotralstat capsules.¹ The company did not report new efficacy data for the pellet formulation.

Efficacy for the 150 mg dose rests on the existing capsule program. Across 2 clinical studies, berotralstat 150 mg capsules produced a mean attack-rate reduction of 91% at 96 weeks in patients aged ≥ 12 years.¹ Rescue medication use fell by 88% across the same studies.¹

The pellets resemble sprinkles in size and appearance. Patients may pour them directly into the mouth and swallow immediately with water or milk, or sprinkle them on a spoonful of soft, non-acidic food.¹ BioCryst positions the formulation for patients with swallowing difficulty, those who prefer to avoid capsules, and adolescents who began pellet therapy as children.¹

For pediatric patients aged 2 to 11 years, the label doses pellets by body weight in unit-dose packets of 72 mg, 96 mg, 108 mg, or 132 mg.³ The new 150 mg pellet matches the established capsule dose for patients aged ≥ 12 years.¹

Berotralstat Safety Profile and Prescribing Considerations

According to BioCryst, berotralstat was safe and well tolerated in both capsule trials. Gastrointestinal reactions and back pain were the most frequently reported adverse reactions versus placebo.¹ The gastrointestinal reactions generally occurred soon after treatment initiation, became less frequent with time, and typically resolved on their own.¹

Adverse reactions occurring in ≥ 10% of patients included abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.¹ QTc interval increases were observed in adults at dosages above 150 mg once daily and were concentration dependent. Additional doses, or doses exceeding the prescribed once-daily dose, are not recommended.¹

Berotralstat is not indicated for the treatment of acute HAE attacks. For patients aged ≥ 12 years with moderate or severe hepatic impairment (Child-Pugh B or C), the recommended dosage is 110 mg capsules once daily with food.¹ Use should be avoided in pediatric patients aged 2 to 11 years with moderate or severe hepatic impairment.¹

Drug interactions also warrant review. Berotralstat is a P-glycoprotein (P-gp) substrate, and concomitant P-gp inducers should be avoided due to the risk of reduced efficacy.¹ At 150 mg, berotralstat moderately inhibits CYP2D6 and CYP3A4, so clinicians should closely monitor or adjust substrates of these enzymes where small concentration increases could cause serious adverse reactions.¹

Berotralstat capsules are approved in > 45 countries.¹ BioCryst supports both formulations through Empower Patient Services, which combines a specialty pharmacy with insurance, affordability, and adherence assistance.¹

References

1. BioCryst Pharmaceuticals. BioCryst announces U.S. FDA approval of ORLADEYO 150 mg oral pellets for adults and adolescents with HAE. Published October 21, 2026. Accessed October 22, 2026. [INSERT LIVE GLOBENEWSWIRE URL]

2. BioCryst Pharmaceuticals. BioCryst announces FDA approval of ORLADEYO (berotralstat) oral pellets, first and only oral prophylactic treatment for patients with HAE aged 2 to <12 years. Published December 12, 2025. Accessed October 22, 2026. https://ir.biocryst.com/node/27561

3. Orladeyo (berotralstat). BioCryst Pharmaceuticals; revised December 2025. Accessed October 22, 2026. https://www.biocryst.com/wp-content/uploads/sites/13/2025/12/ORLADEYO_USPI.pdf