Oral Berotralstat as effective as intravenous agents for long-term prophylaxis of hereditary angioedema

Hereditary angioedema (HAE) is a rare genetic condition that causes swelling under the skin and lining of the gut and lungs. It can happen in different parts of our body. Berotralstat, a first-line, once-daily oral plasma kallikrein inhibitor for long-term prophylaxis of HAE, is an effective and well-tolerated treatment option.

A study report summarizes the safety, effectiveness, and impact on treatment satisfaction in patients who switched from injectable long-term prophylactics (LTPs) to oral berotralstat monotherapy (150 mg daily) at US sites in the international open-label APeX-S study. The study is published in Annals of Allergy, Asthma & Immunology.

The study finds that injectable prophylactic medication to berotralstat was safe and tolerable. Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction.

Researchers in APeX-S (ClinicalTrials.gov, NCT03472040) an open-label Phase 2 study of berotralstat conducted in 22 countries. They focused on APeX-S patients enrolled at US sites who switched from injectable LTPs to berotralstat 150 mg once-daily monotherapy.

The key findings of the study are

• Thirty-four patients discontinued lanadelumab (n = 21), subcutaneous C1 esterase inhibitor (n = 11), or intravenous C1 esterase inhibitor (n = 2) and switched to berotralstat 150 mg monotherapy.

• Vomiting, diarrhea, and upper respiratory tract infection were the most common adverse events (each 11.8%). Mean monthly attack rates were consistently low following the switch to berotralstat.

• The mean (standard error of the mean) monthly attack rate was 0.29 (0.11) at Month 1, 0.48 (0.15) at Month 6, and 0.58 (0.23) at Month 12. The median attack rate was 0 attacks/month throughout 12 months of treatment.

• Improvements were observed in the Treatment Satisfaction Questionnaire for Medication from baseline to Month 12 following the switch to berotralstat monotherapy, with the greatest improvements in convenience.

Researchers concluded that “The transition from injectable prophylactic medication to berotralstat was generally well tolerated. Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction.”

Reference: Marc A. Riedl MD MS, Daniel Soteres MD, J. Wesley Sublett MD; Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat; Annals of Allergy, Asthma & Immunology; DOI: https://doi.org/10.1016/j.anai.2023.11.016.