BioCryst Pharmaceuticals Presents New Research on HAE Treatment Burdens at US HAEA National Summit

BioCryst Pharmaceuticals is presenting new research on HAE treatment burdens at the 2025 US HAEA National Summit. The research focuses on pediatric patients under 12 and their caregivers, as well as patient perspectives on HAE treatments. BioCryst's oral therapy, ORLADEYO, offers a once-daily prophylactic option for HAE, potentially improving patient compliance and quality of life. However, the safety and effectiveness of ORLADEYO for acute HAE attacks have not been established, and regulatory approvals for pediatric use are pending.

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) will present new findings from patient-focused research at the 2025 US HAEA National Summit in Baltimore, July 10-13. The company will showcase data on the burden of current treatments for hereditary angioedema (HAE) on pediatric patients under the age of 12 and their caregivers, as well as patient perspectives on the importance of HAE treatments [1].

The research will be presented in posters at the summit, with abstracts on patient perspectives, the burden of treatment for pediatric patients, and HAE attack rates in pediatric patients aged 2 to 11 years. BioCryst President and CEO Jon Stonehouse will also address members of the HAE community during the event.

BioCryst's oral therapy, ORLADEYO (berotralstat), is a once-daily prophylactic option for HAE, which may improve patient compliance and quality of life. However, the safety and effectiveness of ORLADEYO for acute HAE attacks have not been established, and regulatory approvals for pediatric use are pending [1].

In a separate announcement, KalVista Pharmaceuticals, Inc. (NASDAQ: KALV) recently received FDA approval for EKTERLY (sebetralstat), the first and only oral on-demand treatment for HAE. EKTERLY offers rapid symptom relief and attack resolution, regardless of attack severity, location, age, or use of long-term prophylaxis [2].

These developments highlight the growing focus on patient-centric research and innovative treatments in the management of HAE. Investors and financial professionals should monitor these advancements, as they may impact the companies' market positions and valuation.

References:
[1] https://www.biospace.com/press-releases/biocryst-to-highlight-patient-focused-research-at-the-2025-us-haea-national-summit
[2] https://quantisnow.com/insight/kalvista-pharmaceuticals-announces-fda-approval-of-ekterly%C2%AE-sebetralstat-first-and-only-oral-ondemand-treatment-6111978