Dawnzera (autoinjector)

(Donidalorsen)

Phase 3RNA therapy

Dawnzera (autoinjector) is one of 30 small molecule programs targeting Hereditary Angioedema. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Hereditary AngioedemaActive, not recruiting

Hereditary AngioedemaPhase 3

Active, not recruiting

Competition

Competitive Landscape

43 competitors in Hereditary Angioedema

#DrugCompanyPhaseTrials

Neoral

Crestor

Firazyr

5 4

Lanoxin

Andembry

1 6

Cyklokapron

AAmring

Phase 3

1 7

KALBITOR

Takeda

Phase 3

5 8

C1 Esterase Inhibitor

Takeda

Phase 3

8 9

Ruconest

PPharming Technologies B.V.

Approved

1 10

Orladeyo

BBioCryst Pharmaceuticals

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 3Active, not recruiting

Hereditary Angioedema

NCT05392114

Q2 2029

Phase 3Recruiting

Hereditary Angioedema

NCT07298447

Clinical

Trials by Indication[1]

Hereditary Angioedema6 trials

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

Completed· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 3·NCT05139810·91 enrolled

Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

Available· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals··NCT06415448

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Completed· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 2·NCT04030598·23 enrolled

Show 3 more trials

Donidalorsen Treatment in Children With Hereditary Angioedema

Recruiting· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 3·NCT07298447·20 enrolled

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Completed· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 2·NCT04307381·20 enrolled

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Active, not recruiting· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 3·NCT05392114·154 enrolled

Tics1 trial

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers

Completed· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 1·NCT03263507·32 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 21, 2025

DAWNZERA (AUTOINJECTOR)

NDA NDA219407

Sources

References

[1]
ClinicalTrials.gov — 7 trial records for Donidalorsen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Donidalorsen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Donidalorsen. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA219407). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Dawnzera (autoinjector). U.S. Food & Drug Administration.
[6]
SEC EDGAR — Ionis Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: RNA therapy[2]
Highest Phase: Phase 3[1]
Company: Ionis Pharmaceuticals

Patent Landscape[5]

Patent Cliff

Oct 21, 2032

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9315811Oct 21, 2032

9670492Aug 28, 2034

10294477May 1, 2035

Dawnzera (autoinjector)

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q2 2029

Trials by Indication[1]

Hereditary Angioedema6 trials

Tics1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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