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BioCryst Announces U.S. FDA Approval of ORLADEYO® 150 mg Oral Pellets for Adults and Adolescents with HAE

BCRX
  • The FDA approved BioCryst Pharmaceuticals' supplemental New Drug Application for once-daily ORLADEYO (berotralstat) 150 mg pellet formulation for prophylactic therapy in adults & adolescents aged 12 years & older.
  • This approval expands ORLADEYO as the first & only targeted oral prophylactic therapy option for hereditary angioedema (HAE) patients aged 2 & above, offering a new administration choice for those who struggle with swallowing or prefer pellets over capsules.
  • The pellet formulation is sprinkle-like & can be swallowed directly with water or milk, or sprinkled over soft, non-acidic food.
  • Clinical data from two studies showed ORLADEYO capsules (150 mg) achieved a 91% mean attack-rate reduction at 96 weeks & an 88% reduction in rescue medication use in patients 12 years & older.
  • Common adverse reactions included gastrointestinal issues & back pain, which typically occurred early in treatment & resolved over time.
  • FDA approval was based on chemistry, manufacturing, controls, & data demonstrating the safety & tolerability of the pellet formulation compared to capsules.
  • ORLADEYO capsules were previously approved in December 2020 for patients 12 & older & the pellet formulation received prior approval in December 2025 for pediatric patients aged 2 to <12 years.
  • Safety warnings indicate QTc interval prolongation risks at dosages higher than 150 mg once daily, & the drug is not indicated for treating acute HAE attacks.
  • Dosage adjustments are required for patients aged 12 & older with moderate or severe hepatic impairment (110 mg once daily with food), & use is avoided in pediatric patients aged 2 to <12 years with such impairment.
  • Concomitant use of P-gp inducers is contraindicated, & caution is advised when combining ORLADEYO with CYP2D6 or CYP3A4 substrates due to potential increased exposure & adverse reactions.
  • BioCryst continues to offer Empower Patient Services to assist patients & healthcare providers with insurance navigation, affordability, & adherence support.