BioCryst Shares Breakthrough Data at Important Symposium

New Insights from BioCryst Pharmaceuticals

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) has made significant strides in the treatment of hereditary angioedema (HAE) with the presentation of new findings from the APeX-N trial. These insights were shared at the 7th Bradykinin Symposium held recently, showcasing the importance of collaboration between healthcare providers and HAE patients for improved patient outcomes.

APeX-N Trial Findings

The APeX-N trial is a multi-center observational study carried out across several European countries, aimed at evaluating the safety and overall quality of life of patients undergoing treatment with berotralstat. In this interim analysis involving 56 patients from countries such as the United Kingdom, France, Germany, and Sweden, notable results were observed. Non-serious gastrointestinal issues were reported by 12.5% of participants, emphasizing the importance of monitoring side effects during the treatment.

Patient Response and Adherence

A total of four patients discontinued treatment, primarily due to responses that were less than satisfactory. Importantly, one patient who experienced a severe HAE attack continued on the treatment. Dr. Sorena Kiani, a leading immunologist, noted that the results from the APeX-N trial resonate well with previously documented clinical and real-world evidence, validating berotralstat's role as a pioneer in oral prophylactic treatment for HAE.

Shared Decision-Making in Treatment

A key focus of one of the presentations at the symposium was the relationship between healthcare providers and patients. A dedicated study conducted in Germany delved into the shared decision-making process (SDM) between healthcare providers (HCPs) and patients with HAE. This study highlighted numerous barriers that exist in effective communication and collaboration regarding treatment decisions.

Barriers and Recommendations

Through structured interviews and focus group discussions with HCPs and HAE patients, the need for enhanced HCP awareness of patient experiences was underscored. The findings suggest a stronger emphasis on comprehensive education concerning HAE management could significantly improve treatment outcomes and quality of life for patients.

About ORLADEYO

ORLADEYO® (berotralstat) stands out as the only oral therapy specifically designed to protect patients from HAE attacks, catering to both adults and pediatric patients aged 12 and above. Just one capsule each day plays a crucial role in minimizing the frequency of attacks by inhibiting the activity of plasma kallikrein.

Safety Information and Use

While ORLADEYO is designed for long-term prophylaxis against HAE attacks, its effectiveness in treating acute HAE episodes is yet to be established. Dosage recommendations advise against exceeding 150 mg daily to avoid QT prolongation risks. Patients with moderate to severe hepatic impairment may require a reduced dosage for optimal safety.

Connect with BioCryst Pharmaceuticals

BioCryst Pharmaceuticals remains dedicated to advancing treatment options for rare diseases through innovative research and development. With a commitment to patient care and an impressive pipeline of therapeutic options, BioCryst continues to shape the future of HAE treatment. For more information, individuals can reach out via email or visit their company website.

Frequently Asked Questions

What is the primary indication for ORLADEYO?

ORLADEYO is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older.

What were the key findings of the APeX-N trial?

The APeX-N trial reported a 12.5% occurrence of non-serious gastrointestinal events among participants, reinforcing berotralstat's effectiveness as an oral prophylactic treatment.

How does shared decision-making benefit HAE patients?

Shared decision-making improves communication between healthcare providers and patients, leading to better treatment adherence and enhanced patient quality of life.

What are the recommended dosages for ORLADEYO?

The recommended dosage for ORLADEYO is 150 mg once daily. Adjustments may be necessary for patients with significant hepatic impairment.

How can patients report adverse reactions to ORLADEYO?

Patients can report suspected adverse reactions by contacting BioCryst Pharmaceuticals or visiting the FDA's MedWatch website.

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