Analysis Group Researchers Co-Investigate the Real-World Effects of Berotralstat on Hereditary Angioedema Attack Rates
February 4, 2026
Hereditary angioedema (HAE) is a rare genetic disorder that can cause painful and sometimes fatal episodes of tissue swelling. While patients with HAE can experience substantial clinical and economic burdens related to their disease, berotralstat (an oral long-term prophylactic treatment) has been clinically shown to prevent HAE attacks and prior studies have associated it with reduced HAE attack rates.
To further explore this topic, an Analysis Group team led by Principal François Laliberté, Manager Sean MacKnight, and Associate Ramya Ramasubramanian collaborated with researchers from BioCryst; ENT and Allergy Associates; the University of California, Los Angeles; and Allergy and Asthma Research Associates on a first-of-its-kind study analyzing self-reported HAE attack rates before and after berotralstat initiation, stratified by patients’ HAE type and baseline attack frequency. The analysis used specialty pharmacy data from Optime Care, Inc., the sole dispenser of berotralstat in the US. In an article on their research, the authors detail their methods for comparing monthly attack rates after versus before berotralstat initiation using regression models and assigning HAE type based on laboratory values. They conclude that “people diagnosed and living with HAE had fewer HAE attacks after starting berotralstat,” regardless of the frequency of attacks that patients were experiencing before starting berotralstat.