Drugs / Donidalorsen
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed angioedema, hereditary, 4 2 milestones | |||||
| Filed | US (FDA) | — | angioedema, hereditary, 4 | 2024-11-04 | “the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen” ionis.com ↗ |
| Filed | EU (EMA) | DAWNZERA | angioedema, hereditary, 4 | — | “Donidalorsen is currently under regulatory review in the EU” ionis.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Dawnzera | — | 2026-01-19 | europa.eu ↗ |
| Approved | US (FDA) | DAWNZERA (AUTOINJECTOR) | — | 2025-08-21 | fda.gov ↗ |
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03263507 | Aug 2017 → Oct 2018 | — | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 22 trials · 1 met primary | ||||||
| Phase 2 | NCT04307381 | Mar 2020 → Jan 2025 | hereditary angioedema | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04030598 | Jan 2020 → Jan 2021 | hereditary angioedema | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 33 trials · 1 met primary | ||||||
| Phase 3 | NCT07298447 | Apr 2026 → Jun 2029 expected | hereditary angioedema | Ionis Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT05392114 | Jul 2022 → Dec 2026 expected | hereditary angioedema | Ionis Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05139810 | Dec 2021 → Nov 2023 | hereditary angioedema | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Comparator or background therapy2 trials | ||||||
| Phase 2 | NCT04549922 Comparator | Oct → Dec 2020 | COVID-19 | Hospital do Coracao | Completed | No outcome recorded |
| — | NCT06415448 Background | — | hereditary angioedema | Ionis Pharmaceuticals, Inc. | Available | No outcome recorded |
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2026-01-21 | Ionis Pharmaceuticals | Regulatory DAWNZERA™ (donidalorsen) approved in the European Union for hereditary angioedema (HAE) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced that the European Commission (EC) has approved DAWNZERA™ (donidalorsen) in the European Union (EU) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. |
| 2025-11-14 | Ionis Pharmaceuticals | Regulatory DAWNZERA™ (donidalorsen) receives positive opinion from CHMP, recommended for approval in EU for hereditary angioedema (HAE) ionis.com ↗ |
| 2025-11-06 | Ionis Pharmaceuticals | Results Ionis presents new data demonstrating long-term disease control with DAWNZERA™ (donidalorsen) at ACAAI 2025 Annual Meeting ionis.com ↗
DAWNZERA showed durable efficacy, demonstrating 94% overall mean HAE attack rate reduction at one year in OASISplus open-label extension study |
| 2025-08-21 | Ionis Pharmaceuticals | Regulatory DAWNZERA™ (donidalorsen) approved in the U.S. as first and only RNA-targeted prophylactic treatment for hereditary angioedema ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. |
| 2025-07-21 | Ionis Pharmaceuticals | Results Phase 3 OASISplus data demonstrating benefit of donidalorsen for HAE patients who switched from other prophylactics published in JACI In Practice ionis.com ↗
Results indicate that patients who switched to donidalorsen from prior prophylactic treatments showed a 62% further reduction in mean monthly HAE attack rate from baseline, and 84% of patients surveyed reported preference for donidalorsen. |
| 2025-02-20 | Ionis Pharmaceuticals | Results New positive donidalorsen data to be presented at AAAAI/WAO Joint Congress highlight sustained HAE attack rate reduction and disease control ionis.com ↗
Data also show significant and clinically meaningful improvements in quality-of-life measures in new analyses from Phase 3 and Phase 2 clinical program |
| 2024-11-04 | Ionis Pharmaceuticals | Regulatory Ionis announces FDA acceptance of New Drug Application for donidalorsen for prophylactic treatment of HAE ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. |
| 2024-05-31 | Ionis Pharmaceuticals | Results Ionis presents positive results from OASIS-HAE and OASISplus studies of investigational medicine donidalorsen in patients with hereditary angioedema ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from the Phase 3 OASIS-HAE and OASISplus studies of donidalorsen in patients with hereditary angioedema (HAE) demonstrating significant and sustained reduction in mean monthly HAE attack rates and continued attack rate improvement of >90% with one year of treatment for both monthly or every two-month dosing. |
| 2024-01-22 | Ionis Pharmaceuticals | Results Ionis announces positive topline results from Phase 3 OASIS-HAE study of investigational donidalorsen in patients with hereditary angioedema ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results for the Phase 3 OASIS-HAE study of donidalorsen in people with hereditary angioedema (HAE). |
| 2023-11-09 | Ionis Pharmaceuticals | Results Ionis presents positive two-year results from the Phase 2 open label extension study of donidalorsen in patients with hereditary angioedema ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from an ongoing Phase 2 open-label extension (OLE) study evaluating the safety and efficacy of its investigational prophylactic medicine, donidalorsen, in patients with hereditary angioedema (HAE), a rare and life-threatening genetic disease. |
| 2023-06-01 | Ionis Pharmaceuticals | Results Ionis announces positive donidalorsen late-stage clinical progress in HAE ionis.com ↗
Topline two-year open-label extension (OLE) results continue to demonstrate consistent efficacy and safety, with an overall sustained mean reduction in HAE attack rates of 96% from baseline through two years across dosing groups. |
| 2023-02-21 | Ionis Pharmaceuticals | Results Ionis announces new donidalorsen data and presentations at the 2023 American Academy of Allergy, Asthma & Immunology Annual Meeting ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced additional positive interim data from a Phase 2 open-label extension (OLE) study of donidalorsen, an investigational antisense medicine for the treatment of patients with hereditary angioedema (HAE). |
| 2022-11-13 | Ionis Pharmaceuticals | Results Ionis presents positive Phase 2 data from open label extension study of donidalorsen at 2022 ACAAI Annual Meeting ionis.com ↗
Interim data after all patients completed 1 year of treatment in the study showed a sustained reduction in HAE attacks and no new safety signals following treatment with donidalorsen. |
| 2022-03-16 | Ionis Pharmaceuticals | Results Ionis announces publication of positive Phase 2 data for donidalorsen in New England Journal of Medicine ionis.com ↗
In the Phase 2 study, donidalorsen demonstrated a 90% reduction in angioedema attacks compared with placebo at the 80 mg monthly dose. |
| 2021-11-07 | Ionis Pharmaceuticals | Results Phase 2 study results of Ionis' novel antisense treatment for hereditary angioedema to be presented at ACAAI annual meeting ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today that positive results from the Phase 2 study of its investigational antisense medicine, donidalorsen (formerly IONIS-PKK-LRx), will be presented at the American College of Asthma, Allergy & Immunology (ACAAI) Annual Scientific Meeting in New Orleans and via livestream, November 4-8. |
| 2021-03-29 | Ionis Pharmaceuticals | Results Ionis reports positive topline Phase 2 study results of its novel antisense treatment for hereditary angioedema ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that data from a Phase 2 clinical study of IONIS-PKK-LRx met its primary and secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with hereditary angioedema (HAE) compared to placebo. |
| 2020-09-02 | Ionis Pharmaceuticals | Results New England Journal of Medicine publishes study results evaluating Ionis antisense therapy in treatment of patients with hereditary angioedema ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced the publication today of the results from a compassionate-use study evaluating IONIS-PKKRx and IONIS-PKK-LRx in patients living with severe bradykinin-mediated angioedema in The New England Journal of Medicine (NEJM). |
All press releases naming this drug 23 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Donidalorsen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06415448 ↗ |
| Known as | IONIS-PKK-LRx | “A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)” NCT05139810 ↗ |
| Known as | ISIS 721744 | “A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)” NCT05139810 ↗2“Patients were randomized to either one subcutaneous dose of ISIS721744, an antisense that blocks prekallikrein, or placebo.” PMID 39096659 ↗ Aug 2024 |
| Action | Inhibit | “an Antisense Oligonucleotide Inhibitor of PKK” NCT03263507 ↗ |
| Modality | Antisense oligonucleotide | “an Antisense Oligonucleotide Inhibitor of PKK” NCT03263507 ↗ |
| Route | Subcutaneous | “Administered Subcutaneously” NCT03263507 ↗ |
| Target | PKK | “an Antisense Oligonucleotide Inhibitor of PKK” NCT03263507 ↗ |