BioCryst Pharmaceuticals announced the enrollment of the first participant in the pivotal APeX-P trial, which is designed to evaluate the pharmacokinetics and safety of berotralstat (Orladeyo®) treatment for the prevention of hereditary angioedema (HAE) attacks in pediatric participants who are 2 to less than 12 years of age.

The new pediatric formulation of the oral, once-daily treatment “could significantly reduce the treatment burden for children and families impacted by HAE,” said Ryan Arnold, PhD, chief medical officer of BioCryst, in a press release.

APeX-P is a sequential, 3-part, single-arm, open-label study in which participants will receive oral berotralstat once daily at a weight-based dose in up to 4 cohorts. Researchers plan to enroll 20 participants.

Continue Reading

Read about HAE experimental therapies

First, higher-weight cohorts (cohorts 1 and 2) will enroll in parallel. Safety and pharmacokinetics data collected from the 2 first cohorts will then be used to confirm the weight bands for cohorts 3 and 4, which will enroll sequentially.

The trial will consist of 2 treatment periods, starting with a 12-week standard-of-care treatment period, followed by a 48-week berotralstat treatment period, with continuation up to 144 weeks.

The primary endpoint of APeX-P is the characterization of berotralstat’s pharmacokinetics. Secondary and other endpoints are the frequency and severity of adverse events, serious adverse events, and hereditary angioedema attacks, and the acceptability/palatability of the medication.

The study is estimated to be completed by July 2027. Following the trial, BioCryst plans to submit a supplemental New Drug Application to the US Food and Drug Administration for an additional indication for berotralstat for preventing attacks in children and adolescents with HAE.


BioCryst begins enrollment in pivotal APeX-P trial evaluating ORLADEYO® (berotralstat) in pediatric patients with hereditary angioedema. News release. BioCryst Pharmaceuticals, Inc.; January 26, 2023.

Berotralstat treatment in children with hereditary angioedema (APeX-P). July 12, 2022. Updated November 25, 2022. Accessed January 30, 2023.