BioCryst Unveils New ORLADEYO® (berotralstat) Data at 7th Bradykinin Symposium

BioCryst Pharmaceuticals, Inc. has unveiled six posters at the 7th Bradykinin Symposium in Berlin, presenting pivotal data from the APeX-N trial and emphasizing the significance of shared decision-making (SDM) in managing hereditary angioedema (HAE). These presentations, held from September 5-6, 2024, highlighted the benefits of SDM between healthcare providers (HCPs) and HAE patients to ensure optimal patient outcomes.

APeX-N Interim Results: Oral Berotralstat for HAE Prophylaxis in Europe

The APeX-N trial is a multi-center observational study across Europe that evaluates the safety and effectiveness of berotralstat 150 mg in regular clinical settings. This interim analysis encompassed 56 patients from the United Kingdom, France, Germany, and Sweden. The study's primary objective is to assess safety, while secondary objectives include evaluating effectiveness and quality of life.

The interim results revealed that non-serious gastrointestinal adverse events occurred in 12.5% of the patients. Four patients (7%) discontinued treatment, with three citing unsatisfactory responses and one opting to participate in a clinical trial. Notably, one patient experienced a severe HAE attack but continued with the treatment. Dr. Sorena Kiani, a consultant immunologist at Royal Free London NHS Foundation, remarked that these findings reinforce the efficacy of berotralstat as the first oral prophylactic treatment for HAE.

HCP and Patient Perspectives: HAE Long-Term Prophylaxis and Shared Decision-Making

A study conducted in Germany explored the dynamics between HCPs and patients in managing HAE, focusing on identifying barriers to SDM and strategies to enhance it. The study involved ten HCPs who participated in hour-long interviews and simulated patient consultations, as well as eight HAE patients who partook in 30-minute interviews. These participants later took part in structured focus groups to discuss their insights.

The study highlighted the need for improved HCP awareness of patient perspectives, more comprehensive conversations between HCPs and patients, and better education regarding HAE treatment management. Dr. Emel Aygören-Pürsün from the University Hospital Frankfurt underscored the importance of these findings in developing future guidance and strategies for HAE management to facilitate effective shared decision-making and enhance patient quality of life.

About ORLADEYO® (Berotralstat)

ORLADEYO® (berotralstat) is the first and only oral medication specifically designed to prevent HAE attacks in patients aged 12 and older. This once-daily capsule functions by reducing plasma kallikrein activity, thereby preventing HAE episodes.

U.S. Indication and Important Safety Information

ORLADEYO® is indicated for prophylaxis to prevent HAE attacks in adults and pediatric patients aged 12 years and older. However, it is not intended for the treatment of acute HAE attacks, and higher doses than the recommended 150 mg once-daily dosage are not advised due to the potential for QT prolongation. Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

For patients with moderate or severe hepatic impairment, a reduced dosage of 110 mg taken once daily with food is recommended. Additionally, ORLADEYO interacts with several other medications, necessitating appropriate monitoring and dose adjustments.

About BioCryst Pharmaceuticals

BioCryst Pharmaceuticals is a global biotechnology firm committed to enhancing the lives of individuals with complement-mediated and other rare diseases. The company leverages its expertise in structure-guided drug design to develop innovative oral small-molecule and protein therapeutics for challenging diseases. BioCryst has successfully commercialized ORLADEYO® and is advancing a pipeline of novel therapies.

In conclusion, the presentations at the Bradykinin Symposium underscore the promising results of berotralstat in HAE management and the crucial role of shared decision-making in optimizing patient care. These insights are poised to shape future strategies and improve the quality of life for HAE patients.

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