Drugs / Volanesorsen
Volanesorsen
also known as
ISIS 304801 · IONIS-APOCIIIRx · ISIS 304801 FREE ACID · ISIS 304801 SODIUM SALT · ISIS-304801 SODIUM · VOLANESORSEN SODIUM · Waylivra
Antisense oligonucleotide targets APOC3 via inhibition
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed familial chylomicronemia syndrome 3 milestones | |||||
| Filed | US (FDA) | — | familial chylomicronemia syndrome | 2017-08-31 | “today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for volanesorsen” ionis.com ↗ |
| Filed | EU (EMA) | — | familial chylomicronemia syndrome | 2017-07-27 | “a marketing authorization application (MAA) has been submitted to the European Medicines Agency (EMA) for volanesorsen for the treatment of patients with familial...” ionis.com ↗ |
| Filed | Other | — | familial chylomicronemia syndrome | — | “Volanesorsen filings for marketing approval have been submitted in the U.S., EU and Canada.” ionis.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved · conditional | EU (EMA) | Waylivra | — | 2019-05-03 | europa.eu ↗ |
Trials 8
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02910635 | Sep → Dec 2016 | — | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT02639286 | Dec 2015 → Sep 2019 | partial lipodystrophy | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Completed | No outcome recorded |
| Phase 2 | NCT01529424 | Feb 2012 → Jan 2014 | hypertriglyceridemia | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2/31 trial · 1 met primary | ||||||
| Phase 2/3 | NCT02527343 | Dec 2015 → Jun 2018 | familial partial lipodystrophy | Akcea Therapeutics | Terminated | Met primary |
| Phase 33 trials · 2 met primary | ||||||
| Phase 3 | NCT02658175 | Dec 2015 → Jan 2020 | familial lipoprotein lipase deficiency, hypertriglyceridemia 1 | Akcea Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT02300233 | Feb 2015 → Jul 2016 | hypertriglyceridemia | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT02211209 | Dec 2014 → Dec 2016 | familial chylomicronemia syndrome | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT03544060 | — | familial chylomicronemia syndrome | Akcea Therapeutics | No longer available | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT01647308 Comparator | Jul 2012 → Jan 2014 | type 2 diabetes mellitus | Ionis Pharmaceuticals, Inc. | Terminated | No outcome recorded Stop: Business |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2019-08-07 | Ionis Pharmaceuticals | Results New England Journal of Medicine publishes results from pivotal study of WAYLIVRA® (volanesorsen) in patients with familial chylomicronemia syndrome ionis.com ↗ |
| 2018-08-27 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Receive Complete Response Letter for WAYLIVRA from FDA ionis.com ↗ |
| 2018-05-10 | Ionis Pharmaceuticals | Regulatory FDA Advisory Committee Votes in Favor of WAYLIVRA for Treatment of Familial Chylomicronemia Syndrome ionis.com ↗ |
| 2017-11-15 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Announce Acceptance of Marketing Applications in U.S., EU and Canada for Volanesorsen for the Treatment of FCS ionis.com ↗ |
| 2017-09-12 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Announce Filing of New Drug Submission for Volanesorsen to Health Canada ionis.com ↗
Health Canada grants priority review for volanesorsen NDS filing |
| 2017-08-31 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Announce Submission of New Drug Application for Volanesorsen to the U.S. FDA ionis.com ↗
The NDA submission for volanesorsen marks another important milestone in potentially bringing volanesorsen to patients with FCS. |
| 2017-07-27 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Announce Submission of Marketing Authorization Application for Volanesorsen to the European Medicines Agency ionis.com ↗
Akcea Therapeutics, Inc. (NASDAQ: AKCA) and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that a marketing authorization application (MAA) has been submitted to the European Medicines Agency (EMA) for volanesorsen for the treatment of patients with familial chylomicronemia syndrome (FCS). |
| 2017-03-06 | Ionis Pharmaceuticals | Results Akcea and Ionis Announce Positive Results from Pivotal Study of Volanesorsen in Patients with Familial Chylomicronemia Syndrome (FCS) ionis.com ↗
Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced that the pivotal Phase 3 APPROACH study of volanesorsen met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronemia syndrome (FCS). |
| 2016-12-19 | Ionis Pharmaceuticals | Results Akcea and Ionis Announce Positive Results from COMPASS Phase 3 Study of Volanesorsen ionis.com ↗
Akcea Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced that the Phase 3 COMPASS study met its primary endpoint. |
| 2016-07-28 | Ionis Pharmaceuticals | Regulatory Akcea Therapeutics Receives Orphan Designation in Europe for Volanesorsen for the Treatment of Familial Partial Lipodystrophy (FPL) ionis.com ↗
Akcea Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the European Commission (EC) has designated volanesorsen as an orphan medicinal product for the treatment of familial partial lipodystrophy (FPL). |
| 2016-06-27 | Ionis Pharmaceuticals | Results Akcea Announces Publication of Positive Volanesorsen Clinical Data Showing Reduced Triglycerides and Improved Insulin Sensitivity in Patients with High Triglycerides and Type 2 Diabetes ionis.com ↗
Results from the study showed that, in addition to reductions in plasma TG and apolipoprotein C-III (ApoC-III), a key factor in TG regulation, volanesorsen improved insulin sensitivity and glucose control. |
| 2015-07-29 | Ionis Pharmaceuticals | Results Positive Clinical Data From Volanesorsen Published in the New England Journal of Medicine ionis.com ↗
The New England Journal of Medicine (NEJM) has published positive clinical results from a Phase 2 clinical study evaluating volanesorsen (formerly ISIS-APOCIIIRx) in patients with very high to severely high triglycerides. |
| 2015-07-06 | Ionis Pharmaceuticals | Regulatory Akcea Therapeutics Receives Orphan Drug Designation from the US FDA for Volanesorsen (ISIS-APOCIII Rx) for the Treatment of Familial Chylomicronemia Syndrome ionis.com ↗
Akcea Therapeutics, a wholly-owned subsidiary of Isis Pharmaceuticals, Inc. (NASDAQ: ISIS), announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS). |
All press releases naming this drug 21 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Volanesorsen | “To determine the effects of volanesorsen (ISIS 304801), a second-generation 2'-O-methoxyethyl chimeric antisense inhibitor of apolipoprotein (apo)C-III, on triglyceride (TG)...” PMID 27271183 ↗ Jun 2016 |
| Known as | IONIS-APOCIIIRx | “The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome” NCT02658175 ↗ |
| Known as | ISIS 304801 | “To determine the effects of volanesorsen (ISIS 304801), a second-generation 2'-O-methoxyethyl chimeric antisense inhibitor of apolipoprotein (apo)C-III, on triglyceride (TG)...” PMID 27271183 ↗ Jun 20164“ISIS 304801-CS7 The APPROACH Open Label Study Volanesorsen (ISIS 304801) An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients With Familial...” NCT02658175 ↗ “300 mg of volanesorsen (ISIS 304801) administered Intravenous (IV) single dose” NCT02910635 ↗ “ISIS 304801 is a second-generation antisense inhibitor of APOC3 synthesis.” PMID 26222559 ↗ Jul 2015 |
| Known as | ISIS 304801 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544994 ↗ |
| Known as | ISIS 304801 SODIUM SALT | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544994 ↗ |
| Known as | ISIS-304801 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544994 ↗ |
| Known as | VOLANESORSEN SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544994 ↗ |
| Known as | Waylivra | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544994 ↗ |
| Action | Inhibit | “selective antisense inhibition of APOC3 synthesis” PMID 26222559 ↗ Jul 2015 |
| Modality | Antisense oligonucleotide | “ISIS 304801 is a second-generation antisense inhibitor of APOC3 synthesis.” PMID 26222559 ↗ Jul 2015 |
| Route | Subcutaneous | “ISIS 304801 Administered Subcutaneously to Patients With Severe or Uncontrolled Hypertriglyceridemia” NCT01529424 ↗ |
| Target | APOC3 | “selective antisense inhibition of APOC3 synthesis” PMID 26222559 ↗ Jul 2015 |