Donidalorsen for the Treatment of Hereditary Angioedema: A Review of Clinical Studies
A new monthly injection reduced hereditary angioedema attacks by 81%, offering a gentler prophylactic alternative to current treatments.
This comprehensive review synthesizes clinical trial data for donidalorsen, a GalNAc-conjugated antisense oligonucleotide that silences prekallikrein mRNA in the liver to prevent hereditary angioedema attacks via a novel subcutaneous prophylactic mechanism. The Phase 3 OASIS-HAE trial demonstrated 81% monthly attack rate reduction for Q4W dosing with sustained benefits and acceptable tolerability through 4-year extension, including in patients switching from existing prophylactic agents.
What the study was
- Study design
- review_of_clinical_trials_phase1_through_3
- Category
- rare_diseases
- Maturity
- Potentially Practice-Changing
- Journal
- Drug Des Devel Ther
Why it surfaced
Comprehensive Phase 1-3 review of a novel mechanism (GalNAc-ASO) for HAE prophylaxis; Phase 3 evidence with up to 4-year extension; rare disease with severe episodic morbidity; monthly SC dosing potentially superior convenience profile vs existing IV options.
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