Berotralstat, to Treat Hereditary Angioedema, Under EMA Review

According to Angioedema News, the European Medicines Agency (EMA) is now reviewing berotralstat as a potential therapy to combat severe inflammation attacks in patients with hereditary angioedema.

Hereditary Angioedema

Hereditary angioedema (HAE) is a genetic condition that results in attacks of severe swelling under the skin. In fact, the symptoms make up the name of the disease; “angio” means pertaining to blood vessels, and “edema” is swelling and inflammation. People with hereditary angioedema have a defect, or mutation, on chromosome 11. As a result, their C1-inhibitor, a type of blood protein that regulates disease control, inflammation, and coagulation, doesn’t work the right way. So peptides called bradykinin build up, causing inflammation.

HAE attacks can happen without any trigger, but can sometimes be motivated by stress or injury. Attacks may cause swelling in the lips, eyes, and cheeks. Swelling in the arms, legs, or hands may cause the extremities to be over 2x their normal size. Swelling in the stomach and intestine can cause a pregnancy-like stomach, as well as extreme nausea and vomiting. Finally, swelling in the throat can make it difficult to breathe. If you, or someone you know, has HAE and is experiencing throat swelling, please contact your doctor.

Learn more about hereditary angioedema.


Berotralstat is a once-a-day oral medication from BioCryst Pharmaceuticals designed to prevent HAE attacks. Both the European Medicines Agency, as well as the U.S. Food and Drug Administration, will consider approval within the next 12 months. This medication blocks kallikrein, an enzyme that helps to produce bradykinin.

The application is based on data collected from two clinical trials. These trials aimed to understand the efficacy and safety of berotralstat in preventing HAE-related swelling.

The data showed that:

  • A low dose of 110 mg berotralstat reduced hereditary angioedema swelling attacks by 30% over 24 weeks. It also reduced the severity of HAE attacks by 36.9%.
  • A high dose of 150 mg berotralstat reduced hereditary angioedema swelling attacks by 44.2% over 24 weeks. It also reduced the severity of HAE attacks by 49.2%.
  • 22.5% of patients taking berotralstat experienced 90% reduction in attack rate.
  • In APeX-S, the 2nd clinical trial which lasted for 12 months, most patients receiving a high berotralstat dose had no attacks for at least 6 months.
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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