A new study evaluating the safety and pharmacokinetics of berotralstat (Orladeyo®) in children with hereditary angioedema (HAE) is now open.

The single-arm, open-label, phase 3 study aims to determine the appropriate weight-based dose of the treatment in patients, aged 2 to 12 years to prevent HAE attacks.

There will be 2 treatment periods during the trial. In the first, participants will receive their standard of care for 12 weeks. This will be followed by an open-label treatment period with berotralstat for up to 144 weeks.

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The primary outcome measure will be pharmacokinetics as measured by the maximum concentration of berotralstat in plasma, the area under the curve of plasma concentration of berotralstat from time 0 to the end of dosing, and the apparent oral clearance of berotralstat.

Secondary outcome measures will be the frequency and severity of adverse events, serious adverse events, and HAE attacks. Investigators will also assess the palatability of berotralstat oral granules using a self-reported taste rating scale.

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The trial, which is sponsored by BioCryst Pharmaceuticals, aims to recruit 20 participants in Canada, Europe, Israel, and the UK weighing at least 12 kg. Recruitment is not open yet. The estimated completion date of the study is July 2027.

HAE is a rare disease caused by a deficiency of C1 inhibitor, which leads to the dysregulation of the amount of kallikrein as well as factor XIIa, and factor XIIf that is physiologically generated. It is characterized by repeated episodes of severe swelling in the limbs, face, intestinal tract, and airway among others.

Berotralstat is an oral treatment to prevent HAE attacks. Berotralstat, which is the active substance of Berotralstat is a plasma kallikrein inhibitor. It has been approved by the US Food and Drug Administration to treat patients with HAE, aged 12 years and older.


Berotralstat treatment in children with hereditary angioedema (APeX-P). US National Library of Medicine. Updated July 12, 2022. Accessed July 26, 2022.