Donidalorsen for the treatment of hereditary angioedema

INTRODUCTION: Hereditary angioedema (HAE) is a rare disorder with a prevalence of approximately 1 in 50,000. Management of HAE brings challenges including the frequency of delivery of the medication, routes of medication delivery, cost of treatment and significant impact on quality of life for patients with HAE. Donidalorsen is the newest FDA-approved long-term prophylaxis medication for HAE that offers substantial benefits over its competitors. AREAS COVERED: A search of the current literature on donidalorsen was conducted using the PubMed and Cochrane libraries. In this review, we discuss the mechanism and clinical efficacy of donidalorsen. We also explore the comparison between donidalorsen and other agents on the market for the long-term prophylaxis of HAE. The safety profile of donidalorsen as well as a discussion on the economic impact of donidalorsen is also reviewed. EXPERT OPINION: Donidalorsen offers a promising option for treatment of HAE, with substantial attack reduction, infrequent dosing, and improved quality of life. Continued long-term safety data are needed, but donidalorsen may become a leading prophylactic option for HAE worldwide.