The aim of this technology assessment, which has been commissioned by NICE through the NIHR SR Programme as part of the NICE Single Technology Appraisal process, is to review the submission received from BioCryst Pharmaceuticals on the clinical and cost-effectiveness of Berotralstat (Orladeyo®) for the prevention of recurrent attacks of hereditary angioedema for maintenance treatment of locally advanced or metastatic urothelial cancer after platinum-based chemotherapy. The main source of clinical evidence submitted by BioCryst Pharmaceuticals , consists of a Phase III, muticentre, double-blind  RCT (APeX-2). The Aberdeen HTA Group acts as the Evidence Review Group (ERG) for this technology appraisal and provides an independent critique of the clinical and cost-effectiveness evidence submitted by the company. Based on the findings of the submission received from BioCryst Pharmaceuticals and the ERG’s critique, NICE will release appropriate clinical guidance. For more information see:


Miriam Brazzelli;