Ionis announces FDA acceptance of New Drug Application for donidalorsen for prophylactic treatment of HAE

Core Insights - Donidalorsen is an investigational RNA-targeted medicine aimed at preventing attacks of hereditary angioedema (HAE) and is set for FDA review with a PDUFA date of August 21, 2025 [1][3] - The drug has shown significant efficacy in clinical trials, with a 96% mean reduction in HAE attack rates maintained over three years in the ongoing Phase 2 open-label extension study [4] - Ionis Pharmaceuticals is preparing for its second independent commercial launch, contingent on FDA approval, and has partnered with Otsuka for commercialization in Europe and Asia Pacific [3][4] Company Overview - Ionis Pharmaceuticals has a history of developing RNA-targeted medicines and currently has five marketed products, focusing on areas of high patient need such as neurology and cardiology [8] - The company aims to deliver innovative therapies for serious diseases, leveraging its expertise in RNA technology and disease biology [8] Industry Context - HAE is a rare genetic condition affecting over 20,000 patients in the U.S. and Europe, characterized by recurrent severe swelling [6] - Current treatments for HAE are limited, and many patients still experience breakthrough attacks, highlighting the need for effective prophylactic options like donidalorsen [3][6]