Patients with hereditary angioedema (HAE) consistently had a lower frequency of attacks while on berotralstat treatment, regardless of previous prophylaxis status, according to a study published in the Annals of Allergy, Asthma & Immunology.

Oral berotralstat monotherapy has emerged as a therapeutic option for patients with HAE. Studies indicate that patients who switched to this medication experienced lower rates of HAE attacks. The authors of the study hence sought to investigate the outcomes of patients with HAE who were treated with berotralstat after receiving other prophylactic therapies previously. 

The authors recruited patients who had a confirmed diagnosis of HAE type I or II. These participants actively received 100 or 150 mg of berotralstat from December 2020 until May 2022 and were stratified by previous prophylaxis. Researchers collected data through a sole-source pharmacy where the patients received their berotralstat treatment. 

Continue Reading

The research team obtained baseline attack rates 90 days prior to berotralstat initiation from the participants of the study. During the period in which participants were on berotralstat therapy, median attacks were calculated over a number of 90-day periods; this was accomplished by averaging each patient’s monthly attack rate. It is important to note that some patients on berotralstat treatment did not experience a single attack.

Read more about HAE treatments

The results demonstrated that patients on prior prophylaxis had a median baseline attack rate of 1.7 per month, which decreased to 0.3 to 0.5 during treatment. In patients who had received lanadelumab, the baseline attack rate of 1.0 per month decreased to 0 to 0.5 during the treatment period. As for patients who had received a subcutaneous C1-inhibitor, the baseline attack rate of 1.7 per month was reduced to 0.7 to 1.0 during treatment. In addition, patients who were never given prophylactic treatment also experienced a reduction in attack rate. 

“Patients previously treated with another prophylactic therapy reported consistently low hereditary angioedema attack rates when treated with once daily berotralstat,” the authors concluded.


Riedl M, Desai B, Tilley A, Johnston D, Wasilewski S, Bernstein J. Consistently low hereditary angioedema attack rates observed with berotralstat regardless of previous prophylaxis: real-world outcomes. Ann Allergy Asthma Immunol. 2022;129(5):S30. doi:10.1016/j.anai.2022.08.590