A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Summary

The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

Timeline

Start

2022-07-13

Primary completion

2026-12

Completion

2027-03

Publications

• Lumry WR, Tachdjian R, Craig T, Karakaya G, Gelincik A, Stobiecki M, Jacobs JS, Gokmen NM, Reshef A, Manning ME, Grammatikos A, Giardino F, Guilarte M, Baptist AP, Bordone L, Newman KB, Treadwell S, Lin T, Yarlas A, Riedl MA, Cohn DM. Donidalorsen for Long-Term Prophylaxis of Hereditary Angioedema Attacks: Results from the OASISplus Open-Label Extension Cohort at Year 1. J Asthma Allergy. 2026 Feb 23;19:592079. doi: 10.2147/JAA.S592079. eCollection 2026.
• Riedl MA, Bernstein JA, Jacobs JS, Craig T, Banerji A, Perego F, Lumry WR, Wedner HJ, Gierer S, Manning ME, Bordone L, Treadwell S, Lin T, Newman KB, Yarlas A, Cohn DM. Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis. J Allergy Clin Immunol Pract. 2025 Sep;13(9):2381-2389.e3. doi: 10.1016/j.jaip.2025.06.018. Epub 2025 Jul 17.

Drugs

Indications