Berotralstat
Berotralstat, sold under the brand name Orladeyo, is a medication used to prevent attacks of hereditary angioedema (HAE) in people aged twelve years and older.The most common side effects include abdominal pain, vomiting, diarrhea, back pain, and heartburn.Berotralstat was approved for medical use in the United States in December 2020, and in the European Union in April 2021.

History
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.

References
External links
"Berotralstat". Drug Information Portal. U.S. National Library of Medicine.
Clinical trial number NCT03485911 for "Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)" at ClinicalTrials.gov