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BioCryst presents new long-term, real-world data on ORLADEYO
The Fly

BioCryst presents new long-term, real-world data on ORLADEYO

BioCryst Pharmaceuticals announced new data from the APeX-S and APeX-2 clinical trials which evaluated oral, once-daily ORLADEYO for the prophylactic treatment of hereditary angioedema demonstrating sustained reductions in attack rates and improvement in quality of life among patients living with HAE, highlighting its profile as a well-tolerated, effective and convenient prophylactic HAE therapeutic option. The data are being presented at the 2023 American Academy of Allergy, Asthma & Immunology, AAAAI, annual meeting, which is being held in San Antonio, Texas, from February 24-27, 2023. BioCryst AAAAI 2023 Presentation Highlights: Overall, treatment-emergent adverse events reported in APeX-2 and APeX-S were mild and transient, indicating that ORLADEYO was generally well tolerated. Real-World Outcomes in Patients with Hereditary Angioedema Treated with Berotralstat: This analysis assessed patient-reported HAE attack rates of HAE Type I or Type II patients on ORLADEYO 110 mg or 150 mg QD for at least 120 days based on review of data from a sole-source pharmacy from December 2020 to May 2022. Overall, attack rates subsequently decreased upon initiation with ORLADEYO and remained consistently low through 360 days on therapy. The median reported attack rate was less than or equal to 0.5 attacks/month across all reporting periods through 360 days, and the median reported attack rate was 0.0 in half of the 30-day reporting periods. Disease and Treatment Burden of Hereditary Angioedema in Pediatric Patients: Assessment by Caregivers: Only 17 percent of pediatric patients were on prophylactic HAE treatment. 40 percent of caregivers reported greater than or equal to 1 attack experienced by the pediatric patient in the past six months. 75 percent of school-age pediatric patients who had greater than or equal to1 attack missed at least one day of school in the last six months because of HAE and 33 percent missed 6-15 days. Caregivers of pediatric patients who had greater than or equal to1 attack not on prophylactic therapy most commonly reported infusion requirements and administration inconvenience as reasons why pediatric patients were not on prophylactic therapy. Long-term HAE Prophylaxis with Berotralstat Is Well Tolerated and Effective: Analysis for the APeX-S Study: This analysis characterizes the final safety and effectiveness results of APeX-S through 96 weeks. No new safety signals were observed. Overall, TEAEs were generally mild and transient, indicating that ORLADEYO was generally well tolerated. Berotralstat Improved Quality of Life through 96 Weeks Across Multiple Subgroups of Patients with Hereditary Angioedema; This analysis focused on the changes in QoL assessed in APeX-2 using the validated patient-reported Angioedema Quality of Life Questionnaire. Mean patient-reported improvements from baseline to Week 96 in total AE-QoL score exceeded the minimal clinically important difference value starting at Week 4 and were sustained through Week 96. Improvements were also observed in all domains of the AE-QoL regardless of stratification, with the largest improvement occurring in the functioning domain in almost all stratification groups.

Published first on TheFly

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