Berotralstat for Hereditary Angioedema

(APeX-A Trial)

This trial aims to provide continued access to berotralstat, a medication for people with hereditary angioedema (HAE), a condition that causes sudden and severe swelling. It is intended for those who participated in previous berotralstat studies and found the treatment beneficial. Participants should not use other long-term treatments for HAE attacks. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of this treatment for others.

Yes, you will need to stop taking other medications for long-term prevention of HAE attacks and any other investigational drugs during the study.

Research has shown that berotralstat is generally safe for people with hereditary angioedema (HAE). Long-term studies found that it effectively reduces the number of HAE attacks. Importantly, these studies identified no major safety issues. Participants took berotralstat daily, and it was well-tolerated across all age groups, including children, teenagers, and adults. This consistent safety record makes berotralstat a promising option for managing HAE.¹²³⁴⁵

Berotralstat is unique because it offers a once-daily oral option for managing hereditary angioedema (HAE), which is a departure from the injections or intravenous treatments commonly used for this condition. Its mechanism of action targets the kallikrein-kinin system, which helps prevent the excessive buildup of bradykinin, a protein that contributes to swelling in HAE. Researchers are excited about berotralstat because it simplifies treatment with a convenient oral form, potentially improving patient adherence and quality of life.

Research shows that berotralstat helps reduce the number of hereditary angioedema (HAE) attacks. Participants in this trial will receive berotralstat, which studies have shown results in a 29-100% drop in attack frequency. Many also experienced less severe attacks. Studies confirm that patients who switched to berotralstat managed their symptoms well and felt more satisfied with their treatment. Overall, berotralstat has consistently proven safe and effective for people of all ages, leading to a better quality of life for those with HAE.³⁴⁵⁶⁷