BioCryst Pharmaceuticals, Inc. has recently presented new real-world evidence on the use of ORLADEYO (berotralstat), an oral, once-daily treatment for hereditary angioedema (HAE) patients in the United States. The findings demonstrate significant reductions in healthcare resource utilization (HRU) after starting ORLADEYO therapy.
HAE is a rare genetic disorder characterized by recurrent episodes of swelling in various body parts, such as the hands, feet, face, and airways. These attacks can be debilitating and potentially life-threatening if not promptly treated. Traditionally, attenuated androgens have been used as a prophylactic treatment for HAE, but they come with various tolerability issues and long-term safety concerns.
The recent real-world evidence presented by BioCryst showcases the effectiveness of ORLADEYO in reducing attack rates among HAE patients with normal C1-inhibitor levels. The study establishes ORLADEYO as a promising alternative prophylactic option for these patients, who face limited treatment options compared to those with low or undetectable C1-inhibitor levels.
The data reveal that HAE patients with normal C1-inhibitor levels experienced a significant decrease in attack rates after initiating ORLADEYO treatment. This reduction subsequently led to a decrease in HRU, highlighting the impact of this therapy on improving the quality of life for HAE patients.
Furthermore, BioCryst has previously stated that ORLADEYO showed significant reductions in healthcare resource utilization among HAE patients in general. This further emphasizes the compelling evidence showcasing the effectiveness of ORLADEYO as an HAE prophylactic option.
In addition, a publication in JACI: In Practice reports on the open-label extension of the APeX-2 pivotal trial of ORLADEYO. The results demonstrate rapid and sustained reductions in HAE attacks and improved quality of life for patients over a 96-week treatment period. This study further solidifies the long-term efficacy and safety of ORLADEYO in managing HAE.
Overall, these findings provide crucial real-world evidence on the effectiveness of ORLADEYO as a prophylactic treatment for HAE. The reduction in attack rates, improved quality of life, and decreased healthcare resource utilization observed in HAE patients underscore the importance of accessible and safer treatment options for this rare genetic disorder.
In conclusion, BioCryst Pharmaceuticals’ new real-world evidence and publication in JACI: In Practice highlight the significant impact of ORLADEYO on reducing HAE attacks, improving quality of life, and decreasing healthcare resource utilization. These findings underscore the need for accessible and tolerable HAE prophylaxis options, emphasizing ORLADEYO as a promising treatment option for HAE patients.
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