Drug

Donidalorsen

Approved · EMA

Emerging researchLate-stage development

Also known as DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-SP-G-SP-T-SP-M5C-SP-T-SP-M5C-SP-T-SP-T-SP-G-SP-G-SP-M5C-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL), Dawnzera.

RxNorm2724361UNIIZD4D8M32TL

Research papers

Active clinical trials

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema

Clinical trial2026-05-28Results expected Q2 2029 · ClinicalTrials.gov

An Open-Label Extension Study of ISIS 721744 in Patients With Hereditary Angioedema

Clinical trial2026-05-08Results posted · ClinicalTrials.gov

Approval: Dawnzera (EMA)

Regulatory2026-01-19EMA

An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Clinical trial2025-12-12Results expected Q4 2026 · ClinicalTrials.gov

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

Clinical trial2025-03-06Results posted · ClinicalTrials.gov

Efficacy and Safety of Donidalorsen for Hereditary Angioedema.

Research2024-05-31The New England journal of medicine

A Randomized, Double-Blind, Placebo-Controlled, Phase 2a Study to Assess the Clinical Efficacy of ISIS 721744, a Second-Generation Ligand-Conjugated Antisense Inhibitor of Prekallikrein, in Patients With Hereditary Angioedema

Clinical trial2023-04-03Results posted · ClinicalTrials.gov

A Double-Blind, Placebo-Controlled, Dose-Escalation Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 721744, an Antisense Oligonucleotide Inhibitor of PKK, Administered Subcutaneously to Healthy Volunteers

Clinical trial2018-10-09Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Donidalorsen

Aliases & brands

DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-SP-G-SP-T-SP-M5C-SP-T-SP-M5C-SP-T-SP-T-SP-G-SP-G-SP-M5C-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL)Dawnzera

RxNorm CUI

2724361

UNII

ZD4D8M32TL

Regulatory jurisdictions

ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2026-01-19

Latest approval

2026-01-19

Authorities

EMA

Total events

emaEuropean Medicines Agency· 1 event

2026-01-19Approval

Approval: Dawnzera (EMA)

Indication: Dawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.