|Other names||BCX7353, BCX-7353|
|Drug class||Plasma kallikrein inhibitor|
|Main uses||Hereditary angioedema (HAE)|
|Side effects||Abdominal pain, vomiting, diarrhea, back pain, heartburn|
|Typical dose||150 mg OD|
|Chemical and physical data|
|Molar mass||562.573 g·mol−1|
|3D model (JSmol)|
Berotralstat, sold under the brand name Orladeyo, is a medication used to prevent attacks of hereditary angioedema (HAE). It is used in people aged twelve years and older. It is taken by mouth.
Common side effects include abdominal pain, vomiting, diarrhea, back pain, and heartburn. Other side effects may include QT prolongation. While there is no evidence of harm in pregnancy, such use has not been well studied. It is a plasma kallikrein inhibitor.
Berotralstat was approved for medical use in the United States in 2020 and Europe in 2021. In the United Kingdom 4 weeks costs the NHS about £10,200 as of 2022. This amount in the United States costs about 40,500 USD.
It is generally taken at a dose of 150 mg once per day.
Berotralstat was approved based on evidence from one clinical trial (Trial 1 /NCT03485911) of 120 participants with hereditary angioedema. The trial was conducted at 40 sites in the United States, the European Union, and Canada. Trial investigators evaluated participants 12 years and older with hereditary angioedema for eight weeks to determine the number of attacks for each participant. The trial enrolled only participants who had at least two attacks during the eight-week period. Participants were assigned to receive one of two doses of berotralstat or placebo once every day for 24 weeks. Neither the participants nor the investigators knew which treatment was being given until after the trial was completed. All participants could use other medications for treatment of attacks.
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