BioCryst Presents New Data on the Long-term Efficacy and Safety of ORLADEYO® (berotralstat) Across all Ages at EAACI

Core Insights - BioCryst Pharmaceuticals announced new data demonstrating the long-term efficacy and safety of ORLADEYO (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) across all age groups [1][2] Group 1: Efficacy and Safety - Data from clinical trials and real-world settings indicate that ORLADEYO is an effective and well-tolerated long-term prophylactic treatment for HAE, with patients experiencing fewer and less severe attacks [2] - The ongoing APeX-P study shows that berotralstat significantly reduces the number of HAE attacks requiring treatment or professional care in pediatric patients aged 2 to 11 years [4] - In the Berolife study, patients aged over 12 years reported a mean baseline attack rate of 1.1 attacks per month, which decreased significantly during treatment [7][10] Group 2: Quality of Life Improvements - Patients reported an 86% reduction in attacks requiring professional care, with the mean adjusted monthly attack rate decreasing from 1.28 to 0.38 during the initial weeks of treatment [10] - Significant improvements in quality of life were observed, with 60% of patients achieving the Minimal Clinically Important Difference (MCID) in the Angioedema Quality of Life Questionnaire (AE-QoL) total score at week 24 [20] - Participants noted reduced frequency and severity of HAE attacks, leading to improved disease management and a greater sense of normalcy in daily life [21] Group 3: Patient Experience - A qualitative study revealed that 71% of participants had switched to berotralstat from previous treatments due to long-term tolerability concerns [14] - Patients described berotralstat as less burdensome and easier to incorporate into daily routines compared to prior therapies, contributing to enhanced quality of life [21] Group 4: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [16][17] - The recommended dosage is 150 mg once daily, with specific recommendations for patients with hepatic impairment [22]