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Dawnzera (DONIDALORSEN)
Dawnzera works by binding to a specific messenger RNA molecule to prevent the production of a faulty protein that causes hereditary angioedema.
DONIDALORSEN (Dawnzera), marketed by Ionis Pharmaceuticals, is a prophylactic treatment for hereditary angioedema (HAE) attacks. Its key strength lies in its mechanism of action, which targets and binds to a specific mRNA to prevent the production of the faulty protein causing HAE, offering a precise therapeutic approach. The primary risk is the key composition patent expiry in 2028, which could lead to increased competition from generic alternatives.
At a glance
| Generic name | DONIDALORSEN |
|---|---|
| Sponsor | Ionis Pharms Inc |
| Modality | Oligonucleotide |
| Therapeutic area | Pain |
| Phase | FDA-approved |
| First approval | 2025 |
Mechanism of action
Donidalorsen is an 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 and pain in HAE. In patients with HAE, C1‑inhibitor (C1‑INH) deficiency or dysfunction leads to excessive plasma kallikrein activity, bradykinin generation, and angioedema attacks. Donidalorsen lowers PKK concentration, preventing excessive bradykinin production in patients with HAE.
Approved indications
- Prophylaxis for HAE attacks
Common side effects
- Injection site reactions
- Upper respiratory tract infection
- Urinary tract infection
- Abdominal discomfort
- Decrease in Platelet Count
- Increase in Liver Function Tests
- Hypersensitivity Reactions
- Anaphylaxis
- Generalized rash
- Dyspnea
- Chest pain
- Peri-oral swelling
Key clinical trials
- Donidalorsen Treatment in Children With Hereditary Angioedema (PHASE3)
- A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) (PHASE3)
- Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
- An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema (PHASE2)
- OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) (PHASE3)
- A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema (PHASE2)
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers (PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Dawnzera CI brief — competitive landscape report
- Dawnzera updates RSS · CI watch RSS
- Ionis Pharms Inc portfolio CI