BioCryst Pharmaceuticals(BCRX) Newsfilter·2025-02-12 12:00Core Viewpoint - BioCryst Pharmaceuticals has received a recommendation from Infarmed in Portugal for ORLADEYO® (berotralstat) to be used for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older, marking a significant advancement in treatment options for HAE patients in Portugal [1][2]. Group 1: Product Information - ORLADEYO® (berotralstat) is the first and only oral therapy specifically designed to prevent attacks of hereditary angioedema in patients aged 12 years and older, with a recommended dosage of one capsule per day [3]. - The mechanism of action for ORLADEYO involves decreasing the activity of plasma kallikrein, which is responsible for HAE attacks [3]. - The product is indicated for prophylaxis to prevent attacks of hereditary angioedema in both adult and pediatric patients aged 12 years and older [4]. Group 2: Market Access and Regulatory Status - The Infarmed recommendation follows the European Commission's marketing authorization of ORLADEYO in April 2021, and the product is currently licensed in 44 countries [2]. - The approval in Portugal enhances access to modern prophylaxis for HAE patients, providing more options for healthcare providers and potentially improving the quality of life for patients [2]. Group 3: Safety and Usage Information - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it is not recommended for such use [5]. - Dosages higher than 150 mg once daily are not recommended due to the risk of QT prolongation, with common adverse reactions including abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease [6]. - A reduced dosage of 110 mg is advised for patients with moderate or severe hepatic impairment [6].