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Donidalorsen in children with hereditary angioedema
2025-523499-22-00Phase 3Authorised
- Sponsor
- Ionis Pharmaceuticals
- Start
- Apr 2026
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 5
01
Overview
Phase 3, open-label, single-arm trial of donidalorsen in hereditary angioedema (HAE), sponsored by Ionis Pharmaceuticals. Primary endpoints: Incidence and severity of treatment-emergent adverse events and Plasma pharmacokinetics and concentrations of donidalorsen. Planned start Apr 2026; status: authorised.
02
Drugs
donidalorsen
03
Conditions
hereditary angioedema (HAE)
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Competitive context
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