BioCryst Wins FDA Approval for ORLADEYO 150 mg Pellets in HAE

BioCryst Pharmaceuticals announced on October 21, 2026, that the U.S. Food and Drug Administration (FDA) approved its supplemental New Drug Application (sNDA) for ORLADEYO® (berotralstat) 150 mg oral pellets for the prevention of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. The approval provides an alternative to capsules for patients who have difficulty swallowing them, prefer another dosage form or wish to continue using pellets after reaching 12 years of age.

The decision expands administration choices for an established oral preventive therapy rather than introducing a new active ingredient or demonstrating superior efficacy over the capsule formulation.

Oral Pellets Expand Administration Options

ORLADEYO is a plasma kallikrein inhibitor that reduces kallikrein activity to help prevent HAE attacks. The newly approved 150 mg pellets are intended for once-daily administration in patients aged 12 years and older.

The pellets can be poured directly into the mouth and swallowed immediately with water or milk. Alternatively, they can be sprinkled over a spoonful of soft, non-acidic food. This provides another way to administer the treatment without requiring patients to swallow a capsule.

The formulation may be particularly relevant for patients who experience difficulty swallowing solid dosage forms. However, the approval announcement does not establish that pellets improve adherence, treatment persistence or clinical outcomes compared with capsules.

Approval Extends an Existing Age-Specific Formulation Strategy

The FDA approved ORLADEYO capsules in December 2020 for HAE attack prevention in patients aged 12 years and older. In December 2025, the agency approved the oral pellet formulation for children aged 2 to under 12 years.

The latest decision adds a 150 mg pellet option for adults and adolescents aged 12 years and older. ORLADEYO therefore offers capsule and pellet formulations for patients in this older age group, alongside weight-based pediatric pellet doses for younger children.

BioCryst describes ORLADEYO as the first and only targeted oral prophylactic therapy for patients with HAE aged 2 years and older. This is the company’s positioning of its treatment portfolio; it should not be interpreted as a claim that the October 2026 decision represents the first approval of ORLADEYO across all these age groups.

Clinical Evidence and Basis for Approval

BioCryst reported that the FDA based its approval on chemistry, manufacturing and controls information and data demonstrating the safety and tolerability of the pellet formulation compared with ORLADEYO capsules.

The company also highlighted previously reported efficacy data from two clinical studies of ORLADEYO capsules at 150 mg once daily in patients aged 12 years and older. At 96 weeks, the mean HAE attack rate was reduced by 91%, while rescue medication use decreased by 88%.

These results provide context for the established efficacy of ORLADEYO, but they are not direct evidence that the newly approved pellets provide greater attack prevention than capsules. The formulation-specific approval was supported by manufacturing and quality information and comparative safety and tolerability data, rather than a reported new pivotal efficacy trial demonstrating superiority.

Safety and Prescribing Considerations

ORLADEYO is indicated for prophylaxis to prevent HAE attacks and should not be used to treat acute attacks. Additional doses or doses above the prescribed once-daily regimen are not recommended because of the risk of QTc interval prolongation.

Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain and gastroesophageal reflux disease. Gastrointestinal reactions may occur early during treatment and can become less frequent over time.

Berotralstat also has clinically relevant drug interactions. Concomitant use with P-glycoprotein inducers should be avoided, while certain medicines metabolized by CYP2D6 or CYP3A4, or transported by P-glycoprotein, may require monitoring or dosage modification. Prescribers should consult the current prescribing information when selecting concomitant therapies.

Implications for HAE Management

The FDA approval broadens the available administration options for patients receiving long-term HAE prophylaxis. By offering 150 mg oral pellets alongside capsules for adults and adolescents, BioCryst can accommodate differing administration preferences without changing the established preventive treatment approach.

The practical significance of this decision will depend on whether patients and prescribers find the alternative formulation useful in routine care. The approval itself does not establish improved adherence, superior efficacy or better tolerability than capsules.

Reference

BioCryst Announces U.S. FDA Approval of ORLADEYO® 150 mg Oral Pellets for Adults and Adolescents with HAE, BioCryst Pharmaceuticals,  October 21, 2026.