BioCryst to Present Data Showing Reduced Medical Visits and Hospitalizations with ORLADEYO®

BioCryst Pharmaceuticals, Inc., headquartered in RESEARCH TRIANGLE PARK, N.C., has unveiled new substantial real-world evidence supporting the efficacy of its oral, once-daily drug, ORLADEYO® (berotralstat), in reducing the healthcare resource utilization (HRU) for patients suffering from hereditary angioedema (HAE) in the United States. The findings indicate marked decreases in hospitalizations, emergency room visits, and the use of on-demand therapies following the initiation of ORLADEYO treatment. These new insights are scheduled to be presented at the Academy of Managed Care Pharmacy (AMCP) Nexus 2024 national meeting in Las Vegas, taking place from October 14 to 17, 2024.

Sandra Christiansen, MD, a professor of medicine and director of translational research at the US HAEA Angioedema Center at the University of California, San Diego, highlighted the significance of these findings. Dr. Christiansen emphasized that the analysis of claims data not only corroborates previous studies on ORLADEYO's positive impact on HAE patients but also underscores its broader benefits to the healthcare system. The recent investigation showcased substantial reductions in healthcare resource utilization across various outcomes, including fewer hospitalizations and medical visits related to HAE attacks.

The study, titled "Healthcare Resource Utilization among Patients Initiating Berotralstat for the Long Term Prophylaxis of Hereditary Angioedema in the United States," drew from a retrospective analysis of U.S. administrative claims data, focusing on patients covered by commercial and public health plans. Participants were those who had started ORLADEYO between December 2020 and December 2022, with at least six months of continuous health plan enrollment before beginning the treatment (n=260).

The results revealed significant reductions in HRU among the overall study cohort after starting ORLADEYO, including a 34% decrease in all-cause hospitalizations and a 14% reduction in outpatient or emergency room visits. Specifically, angioedema-related hospitalizations saw a 52% reduction, and outpatient or emergency room visits were reduced by 44%. Furthermore, the study observed a 51% decline in HAE attack-related visits, driven by substantial decreases in hospitalization (60%) and outpatient or emergency room visits (50%).

When evaluating HAE attack-related visits by the body location of the attack, there were notable reductions, including a 48% decrease in attacks affecting the head and upper airways, a 58% decrease in attacks involving the gastrointestinal system, and a 52% reduction in attacks at unspecified locations. Additionally, there was a 39% decrease in the use of on-demand therapies administered by healthcare professionals among those who had previously relied on such treatments.

The findings will be exhibited in a poster at the MGM Grand Hotel and Convention Center in Las Vegas on Tuesday, October 15, from 5:00-7:00 pm PT, and will be presented on Wednesday, October 16, from 11:30 am-1:00 pm PT.

ORLADEYO® (berotralstat) is notable for being the first and only oral therapy specifically designed to prevent attacks of hereditary angioedema in patients aged 12 and older. The once-daily capsule works by inhibiting the activity of plasma kallikrein, thereby preventing HAE attacks.

BioCryst Pharmaceuticals remains committed to enhancing the lives of individuals with rare diseases through its innovative treatments. The company's dedication to leveraging structure-guided drug design has already resulted in the commercialization of ORLADEYO and continues to drive its advancement of a pipeline of small-molecule and protein therapies.