NDASUBCUTANEOUSSOLUTION
Approved
Aug 2025
Lifecycle
Launch
Competitive Pressure
0/100
Clinical Trials
7
Data completeness
5/8 — Partial
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. First approved in 2025.
Drug data last refreshed 12h ago · 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, reducing plasma kallikrein and subsequent bradykinin production. It is approved for hereditary angioedema (HAE) patients with C1-inhibitor deficiency or dysfunction. The autoinjector formulation enables patient self-administration for prevention of angioedema attacks.
Launch8.9 years to LOE
Recently approved product in launch phase with potential for rapid commercialization in the rare disease HAE market; early-stage team building expected.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (7)
Donidalorsen Treatment in Children With Hereditary Angioedema
Started Apr 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 Mar 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.