Indicated for: hereditary angioedema
Route of administration: oral
Substance: berotralstat (enzyme inhibitor)
ATC: B06AC06 (Blood and blood forming organs | Other hematological agents | Drugs used in hereditary angioedema)
Berotralstat is a medication used for the prevention of recurrent hereditary angioedema (HAE) attacks in patients aged 12 years and older. Hereditary angioedema is a rare genetic condition characterized by recurrent episodes of severe swelling of the skin, mucous membranes, and internal organs, caused by a deficiency or dysfunction of the C1 inhibitor. Berotralstat works by selectively inhibiting plasma kallikrein, thereby reducing bradykinin levels, a substance that contributes to inflammation and edema.
The medication is taken orally, usually once daily, as directed by a doctor. It is important for patients to strictly adhere to the treatment regimen to prevent HAE attacks. The treatment is intended solely for prevention and should not be used for managing acute attacks.
Common side effects include abdominal pain, diarrhea, nausea, fatigue, and headache. In rare cases, severe allergic reactions or elevated liver enzyme levels may occur, necessitating regular monitoring of liver function.
Berotralstat is not recommended for pregnant or breastfeeding women unless the benefits outweigh the risks. Patients should inform their doctor about all medications they are taking to avoid drug interactions. Additionally, patients should be aware of the symptoms of an acute attack and have appropriate treatment available for such situations.