NDASUBCUTANEOUSSOLUTION
Approved
Aug 2025
Lifecycle
Launch
Competitive Pressure
0/100
Clinical Trials
7
Data completeness
6/8 — Strong
DAWNZERA (AUTOINJECTOR) (donidalorsen) by Ionis Pharmaceuticals is aso‑galnac conjugate that causes ribonuclease h1 (rnase h1)‑mediated degradation of pkk mrna through binding to pkk mrna, which results in reduced production of pkk protein. Approved for hereditary angioedema. First approved in 2025.
Drug data last refreshed Yesterday · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
DAWNZERA is a subcutaneous antisense oligonucleotide (ASO) conjugate that degrades prekallikrein (PKK) mRNA to reduce plasma kallikrein and bradykinin production in hereditary angioedema (HAE). By lowering PKK concentration, it prevents excessive bradykinin-mediated angioedema attacks in patients with C1-inhibitor deficiency or dysfunction. This represents a novel RNA-targeting mechanism distinct from direct kallikrein inhibitors.
Growth9.0 years to LOE
Recent FDA approval positions DAWNZERA as a growth-stage asset with commercial infrastructure buildout underway; expect expanded sales and field teams as market penetration increases.
Mechanism of Action
ASO‑GalNAc conjugate that causes ribonuclease H1 (RNase H1)‑mediated degradation of PKK mRNA through binding to PKK mRNA, which results in reduced production of PKK protein. PKK is a pro‑enzyme for plasma kallikrein, which results in the release of bradykinin, a potent vasodilator causing swelling…
Indications (1)
Clinical Trials (7)
Donidalorsen Treatment in Children With Hereditary Angioedema
Started Jul 2026
20 enrolled
Hereditary Angioedema (HAE)
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Started Jul 2022
154 enrolled
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
Started Dec 2021
91 enrolled
Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
Started Apr 2020
20 enrolled
Hereditary Angioedema
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
Started Jan 2020
23 enrolled
Hereditary Angioedema
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.