BioCryst Shows High Adherence and Persistence with ORLADEYO (berotralstat)

BioCryst Pharmaceuticals, Inc. has unveiled new real-world research on the use of the oral, once-daily medication ORLADEYO® (berotralstat). This study revealed high adherence and persistence rates for ORLADEYO, comparable to those observed with other long-term prophylactic (LTP) therapies for hereditary angioedema (HAE). Additionally, the research confirmed significant and sustained reductions in HAE attack rates after beginning ORLADEYO, irrespective of the patients' C1-inhibitor (C1-INH) deficiency status. A patient survey also indicated a preference for oral LTP therapy over injectable options.

Dr. Donald S. Fong, BioCryst’s chief medical officer, highlighted the convenience and effectiveness of ORLADEYO as significant factors that differentiate it from other treatments. He emphasized that these results bolster the evidence supporting ORLADEYO as a highly effective and potentially transformative treatment for many HAE patients. The data from these studies is being presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in Boston.

One of the studies, Poster #R072, investigated adherence and persistence rates among patients who started ORLADEYO (n=90) compared to those on lanadelumab (n=189) and subcutaneous plasma-derived C1-inhibitor (SC-pdC1-INH) (n=78). The findings showed that high adherence and persistence rates were maintained across all three therapies, with ORLADEYO demonstrating rates equivalent to, or even better than, the other two options.

Specifically, 86% of ORLADEYO patients, 91% of lanadelumab patients, and 83% of SC-pdC1-INH patients filled at least two prescriptions during the 12-month follow-up period. Adherence rates at 12 months were 77% for ORLADEYO, 76% for lanadelumab, and 80% for SC-pdC1-INH. Persistence rates were also high, with 71% of ORLADEYO patients remaining persistent compared to 63% for both lanadelumab and SC-pdC1-INH. No significant differences in adherence or persistence were observed among the different LTP therapies.

Another set of studies, represented by Posters #R092 and #R093, evaluated the self-reported HAE attack rates before and after starting ORLADEYO in patients with and without C1-INH deficiency. The results demonstrated that ORLADEYO led to significant and sustained reductions in attack rates for 18 months post-initiation. This included patients who already had good control over their attacks at baseline, further confirming the medication's efficacy in maintaining or improving attack control.

For patients with C1-INH deficiency (n=466), the mean monthly attack rate reduced significantly from baseline levels. Similar reductions were observed in patients without C1-INH deficiency (n=353), showing substantial decreases in attack frequency across various follow-up intervals.

Moreover, Poster #R081 explored patient preferences for LTP treatments through an online survey. The survey revealed that effectiveness in preventing attacks and reducing attack severity were the most valued attributes for treatment. When given a choice between an equally effective oral or injectable LTP, the majority of patients (54%) preferred a daily oral therapy over biweekly or monthly injections. Additionally, 52% favored oral therapy even when injections were required as infrequently as every three months.

These findings underscore the importance of considering patient preferences and convenience in treatment plans. ORLADEYO’s effectiveness and patient adherence rates highlight its potential to play a significant role in the long-term management of HAE. As BioCryst continues to generate real-world evidence, these insights are expected to guide physicians in optimizing treatment recommendations tailored to individual patient needs.