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BioCryst to Present New Real-World Evidence Underscoring the Ongoing Burden of Pediatric Hereditary Angioedema at ISPOR 2026
RESEARCH TRIANGLE PARK, N.C., May 13, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will present new real-world evidence at the 2026 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conference taking place in Philadelphia from May 17–20, 2026.
Studies highlight the significant healthcare and quality-of-life burden associated with hereditary angioedema (HAE) in pediatric patients and their caregivers, demonstrating persistent unmet needs and the importance of optimized disease management to reduce healthcare utilization. Underscoring the role of outcomes research in rare diseases, the research draws on large U.S. insurance claims data and mixed-methods caregiver insights to examine both healthcare resource utilization and the day-to-day impact of pediatric HAE on caregivers.
“Hereditary angioedema places a significant burden on children and the families caring for them, which is often underrecognized,” said Christina G. Kwong, MD, allergist and immunologist, Phoenix Children’s. “Understanding both the healthcare utilization impact and caregiver experience through real-world research is key to improving how we care for these kids.”
BioCryst will present the following two poster presentations at the Pennsylvania Convention Center, Level 200, Exhibit Hall:
Healthcare Resource Utilization (HCRU) in Pediatric Patients with Hereditary Angioedema (HAE) in the United States: A Matched Study Using Large Insurance Claims
Poster EE69; Monday, May 18; 12:30 p.m. - 1:30 p.m. ET
This retrospective cohort study analyzed Komodo claims data from 2016 to 2024, including more than 400 children aged 2 to <12 years with HAE, to evaluate healthcare resource utilization compared with pediatric individuals without HAE. Findings indicate that children with HAE experience greater HCRU than matched controls without HAE, including more frequent outpatient and emergency room visits, a higher likelihood of hospitalization, and longer inpatient stays. Outpatient visit rates were more than three times higher among children with HAE, highlighting the considerable healthcare burden associated with the disease.
A Mixed-Methods Study to Explore the Health-Related Quality of Life Impact on Caregivers of Children with Hereditary Angioedema
Poster PCR61; Monday, May 18; 6:00 p.m. - 7:00 p.m. ET
This mixed-methods study examined the health-related quality of life (HRQoL) of caregivers for children aged 2 to <12 years with HAE and assessed how caregiver HRQoL varies with a child’s HAE attack frequency. Caregivers reported spending an average of more than 20 hours per week providing care, including monitoring symptoms, managing medications and medical appointments, supporting daily activities, and providing emotional support. Study findings demonstrate a range of impacts on caregivers’ daily lives, with greater impacts in HRQoL reported during periods when children experienced more frequent HAE attacks.
“At BioCryst, we are committed to advancing a deeper understanding of hereditary angioedema through rigorous real-world evidence generation to further improve quality of life for our patients,” said Sandeep Menon, Chief Research and Development Officer of BioCryst. “The research presented at ISPOR reflects the unmet need and the lived experience of patients and caregivers, particularly in pediatric populations, and reinforces their need for optimized disease management.”
Abstracts are available to view on the ISPOR Presentations Database.
About BioCryst Pharmaceuticals
BioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (“HAE”) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics for a range of rare diseases. For more information, please visit www.biocryst.com or follow us on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding future results, performance, achievements, plans and expectations, including with respect to BioCryst’s commitment and efforts to improve outcomes for patients with HAE and their caregivers. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: risks related to the development and interpretation of clinical and real-world data; and BioCryst’s ability to implement its commercialization plans and successfully commercialize its products. This list is not exclusive. To see a more comprehensive list of risks, please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements.
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