BioCryst Shows Reduced Healthcare Use in HAE Patients After ORLADEYO® Start

BioCryst Pharmaceuticals, Inc. has unveiled new real-world evidence highlighting the benefits of ORLADEYO® (berotralstat) for patients with hereditary angioedema (HAE) in the United States. The data shows that patients experienced notable reductions in healthcare resource utilization (HRU) after starting treatment with this oral, once-daily medication. This information was presented at the 2024 International Society for Pharmacoeconomics and Outcomes Research conference held in Atlanta from May 5-8, 2024.

Sandra Christiansen, MD, from the US HAEA Angioedema Center at the University of California, San Diego, emphasized that the findings provide strong evidence that ORLADEYO not only reduces HAE morbidity but also serves as a cost-effective management strategy. The study indicates substantial benefits for both patients and healthcare payers, contributing to improved quality of life for those affected by HAE.

The study, titled "Reductions in Real-World Healthcare Resource Utilization Among United States Hereditary Angioedema (HAE) Patients Following Berotralstat Initiation," utilized retrospective analysis of administrative claims data from patients who began ORLADEYO treatment between December 2020 and December 2022. The participants had a minimum of six months of continuous health plan enrollment before commencing ORLADEYO, which included both commercial and public health plans.

The overall study population consisted of 260 patients, with subgroups including 126 patients previously treated with long-term prophylaxis (LTP) and 67 patients who were new to LTP but had a history of on-demand treatment. The median follow-up durations were 12 months for the overall population and 13 months for both subgroups.

Results demonstrated significant reductions in HRU across the board after ORLADEYO initiation. For the overall study population:
- All-cause hospitalizations decreased by 34%
- Outpatient or emergency room visits decreased by 14%
- Angioedema-related hospitalizations decreased by 52%
- Angioedema-related outpatient or emergency room visits decreased by 44%

Similar significant reductions were observed in both subgroups. Among LTP-experienced patients:
- Angioedema-related hospitalizations dropped by 54%
- Outpatient or emergency room visits related to angioedema were reduced by 37%

For LTP-naïve patients with a history of on-demand treatment:
- Angioedema-related hospitalizations decreased by 62%
- Outpatient or emergency room visits decreased by 45%

Dr. William Sheridan, chief development officer and interim chief medical officer of BioCryst, expressed the company’s ongoing commitment to showcasing the value of ORLADEYO. He highlighted that the real-world evidence supports the medication's effectiveness in maintaining and improving disease control for HAE patients, irrespective of their treatment history. Dr. Sheridan also mentioned that BioCryst is keen to present more real-world data on the long-term benefits of ORLADEYO.

ORLADEYO (berotralstat) is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 and older. The once-daily capsule works by inhibiting plasma kallikrein activity to prevent these attacks. It is important to note that ORLADEYO should not be used for acute HAE attacks, and dosages higher than 150 mg once daily may cause QT prolongation.

The most common side effects for patients taking ORLADEYO include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. Additionally, dosage adjustments are recommended for patients with moderate or severe hepatic impairment, and caution is advised when ORLADEYO is taken with other medications due to potential interactions.

BioCryst Pharmaceuticals is dedicated to developing innovative treatments for rare diseases, leveraging their expertise in structure-guided drug design. The company continues to advance a pipeline of therapies aimed at addressing unmet medical needs through first-in-class or best-in-class oral small-molecule and protein therapeutics.