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donidalorsen
- Development stage
- Approved
- Lead developer
- Ionis Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Donidalorsen is an RNA-targeted **ligand-conjugated antisense oligonucleotide** developed by Ionis for the **prophylactic treatment of hereditary angioedema**. It is designed to reduce hepatic production of **plasma prekallikrein**, a key upstream mediator in the kallikrein-kinin pathway that drives bradykinin-mediated angioedema attacks. In clinical studies, subcutaneous donidalorsen given every 4 or 8 weeks reduced hereditary angioedema attack rates and improved quality-of-life measures. The drug was studied as an investigational medicine under the codes ISIS 721744 and IONIS-PKK-LRx, and in the U.S. it has been approved and commercialized by Ionis under the brand name DAWNZERA for patients aged 12 years and older.
- Brand names
- DAWNZERA
- Other names
- donidalorsen sodium
02
Targets
PKK (Plasma prekallikrein (KLKB1))
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