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donidalorsen

Generic: DONIDALORSEN SODIUM·Brand: DAWNZERA (AUTOINJECTOR), Dawnzera

donidalorsen: developed by 1 company · 4 clinical trials tracked · 1 FDA decision.

donidalorsen evidence translation

What we can confirm

1 FDA record · 4 tracked trials

What it means

Source-backed records connect donidalorsen to IONS; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could donidalorsen → IONS affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence1 indications · 0 study sponsors

Related indications

Hereditary Angioedema

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (1)
CompanyPhase
IONIS PHARMACEUTICALS INC (IONS)Approved
Clinical Trials (4)

Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)

NCT06415448·IONS
Available
Expanded AccessIonis Pharmaceuticals, Inc.

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

NCT07298447·Phase 3·IONS
Recruiting

Primary endpoint: Number of Participants with Treatment Emergent Adverse Events (TEAEs)

Interventionalopen20 targetStarted 2026-04Completion 2029-0613 sites · Italy, Poland, Spain, United StatesIonis Pharmaceuticals, Inc.

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

NCT05392114·Phase 3·IONS
Active, not recruiting

Primary endpoint: Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity

Interventionalopen154 targetStarted 2022-07Completion 2027-0350 sites · Belgium, Bulgaria, Canada, France…Ionis Pharmaceuticals, Inc.

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

NCT04307381·Phase 2·IONS
Completed

Primary endpoint: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

Interventionalopen20 enrolledStarted 2020-03Completion 2025-017 sites · Netherlands, United StatesIonis Pharmaceuticals, Inc.
FDA Decisions1 records
Approval1 INDICATIONS AND USAGE DAWNZERA™ is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is a prekallikrein directed antisense oligonucleotide indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. ( 1 )(IONS)FDA ↗
NDA219407Type 1 - New Molecular EntityOrphan DrugStandardSubcutaneousSolution

Frequently asked questions

Who is developing donidalorsen?

donidalorsen is being developed by IONIS PHARMACEUTICALS INC.

How many clinical trials involve donidalorsen?

BioSniper tracks 4 clinical trials involving donidalorsen.

What is donidalorsen's latest FDA decision?

The most recent tracked FDA decision for donidalorsen is Approval for 1 INDICATIONS AND USAGE DAWNZERA™ is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is a prekallikrein directed antisense oligonucleotide indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. ( 1 ), dated August 21, 2025.