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Donidalorsen · Dawnzera, ISIS 721744

Oligonucleotide (Antisense oligonucleotide) · Gene silencing: prekallikrein (RIPK4) · developed by Ionis Pharms

Approved FDA, Aug 2025
IONS Developer
1 Registered trials

What is Donidalorsen?

Donidalorsen is an antisense oligonucleotide conjugate that reduces prekallikrein (PKK) protein production by binding to PKK mRNA and triggering RNase H1-mediated degradation. By lowering PKK levels, it prevents excessive bradykinin generation and angioedema attacks in hereditary angioedema.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as
Dawnzera, ISIS 721744, IONIS-PKK-LRx
Drug type
Oligonucleotide (Antisense oligonucleotide)
Targets
prekallikrein (RIPK4)
Mechanism
Gene silencing: prekallikrein (RIPK4)
Pathways
bradykinin generation, angioedema
Therapy area
Immunology
Developer
Ionis Pharms Inc (IONS)
FDA status
Approved as Dawnzera (August 21, 2025), NDA219407 · FDA label
UNII
ZD4D8M32TL

✅ FDA-approved uses

From the indications section of the FDA label (version of Sep 24, 2025). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

TrialPhaseConditionStatusSponsor
NCT05392114 Phase 3 Hereditary Angioedema Active not recruiting IONS

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Hereditary Angioedema

More from Ionis Pharms

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the IONS page.

Follow IONS, the company behind Donidalorsen

See IONS's upcoming FDA decisions, trial readouts, cash runway and dilution risk, updated every day from SEC, FDA and ClinicalTrials.gov filings.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z

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