Drugs / Olezarsen
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed familial chylomicronemia syndrome 1 milestone | |||||
| Filed | US (FDA) | — | familial chylomicronemia syndrome | 2024-05-07 | “Olezarsen NDA submitted to FDA for FCS; preparing EU regulatory submission” ionis.com ↗ |
| Filed hypertriglyceridemia 2 2 milestones | |||||
| Filed | EU (EMA) | Tryngolza | hypertriglyceridemia 2 | 2026-03-30 | “the European Medicines Agency (EMA) has validated an indication extension application for Tryngolza® (olezarsen) for the treatment of adult patients with severe hypertriglyceridemia” sobi.com ↗ “In March 2026 , the European Medicines Agency validated an indication extension application for olezarsen for the treatment of adults with sHTG” sobi.com ↗ “The European Medicines Agency (EMA) accepted an indication extension application in March for the treatment of adult patients with sHTG” ionis.com ↗ |
| Filed | US (FDA) | — | hypertriglyceridemia 2 | 2026-02-26 | “today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for olezarsen for severe...” ionis.com ↗ “sNDA accepted by the FDA for Priority Review for the treatment of sHTG” ionis.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | TRYNGOLZA (AUTOINJECTOR) | — | 2026-06-24 | fda.gov ↗ |
| Approved | EU (EMA) | Tryngolza | — | 2025-09-17 | europa.eu ↗ |
| Approved | US (FDA) | TRYNGOLZA (AUTOINJECTOR) | — | 2024-12-19 | fda.gov ↗ |
Trials 11
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05579860 | Oct 2022 → May 2023 | — | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 22 trials · 2 met primary | ||||||
| Phase 2 | NCT05355402 | May 2022 → Mar 2023 | atherosclerotic cardiovascular disease, hypertriglyceridemia | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 2 | NCT03385239 | Jan 2018 → Nov 2019 | cardiovascular disorder, hypertriglyceridemia | Akcea Therapeutics | Completed | Met primary |
| Phase 37 trials · 3 met primary | ||||||
| Phase 3 | NCT05681351 | Dec 2022 → Mar 2027 expected | — | Ionis Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05610280 | Nov 2022 → Aug 2024 | atherosclerosis, hypertriglyceridemia | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT05552326 | Aug 2022 → Dec 2024 | — | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT05185843 | Feb 2022 → Jun 2027 expected | familial chylomicronemia syndrome | Ionis Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05130450 | Nov 2021 → Feb 2028 expected | familial chylomicronemia syndrome | Ionis Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05079919 | Oct 2021 → Oct 2024 | — | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT04568434 | Nov 2020 → Jul 2023 | familial chylomicronemia syndrome | Ionis Pharmaceuticals, Inc. | Completed | Mixed |
| Phase not stated1 trial | ||||||
| — | NCT06360237 | — | familial chylomicronemia syndrome | Ionis Pharmaceuticals, Inc. | Approved for marketing | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT02900027 Comparator | Sep 2016 → Apr 2018 | hypertriglyceridemia | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 23
| Date | Issuer | Release |
|---|---|---|
| 2026-06-24 | Ionis Pharmaceuticals | Regulatory TRYNGOLZA® (olezarsen) approved by the FDA as the first and only treatment to reduce triglycerides and the risk of acute pancreatitis in patients with severe hypertriglyceridemia (sHTG) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA® (olezarsen) as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG greater than or equal to 500 mg/dL). |
| 2026-05-26 | Sobi, Inc. | Results New analysis shows Tryngolza® (olezarsen) reduced acute pancreatitis by 85% and triglycerides by 66% in severe hypertriglyceridemia sobi.com ↗
Sobi® (STO: SOBI) today announced results from a new analysis of the pivotal Phase 3 CORE and CORE2 trials, showing Tryngolza® (olezarsen) reduced the relative risk of acute pancreatitis events by 85% (P < 0.001) and reduced triglycerides by 66% in patients with severe hypertriglyceridemia (sHTG) after six months. |
| 2026-04-29 | Ionis Pharmaceuticals | Regulatory Ionis reports first quarter 2026 financial results and highlights progress on key programs ionis.com ↗
Olezarsen sNDA accepted by the FDA for Priority Review; sHTG launch preparations on track |
| 2026-03-30 | Sobi, Inc. | Regulatory EMA validates indication extension application for Tryngolza® (olezarsen) for the treatment of severe hypertriglyceridemia (sHTG) sobi.com ↗
Sobi® (STO: SOBI) today announced that the European Medicines Agency (EMA) has validated an indication extension application for Tryngolza® (olezarsen) for the treatment of adult patients with severe hypertriglyceridemia (sHTG) ≥880 mg/dL (≥10 mmol/L). |
| 2026-02-26 | Ionis Pharmaceuticals | Regulatory Olezarsen sNDA accepted by the FDA for Priority Review for the treatment of severe hypertriglyceridemia (sHTG) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for olezarsen for severe hypertriglyceridemia (sHTG). |
| 2026-02-25 | Ionis Pharmaceuticals | Regulatory Ionis reports fourth quarter and full year 2025 financial results and highlights progress on key programs ionis.com ↗
sNDA submitted for marketing approval in U.S. |
| 2026-01-12 | Ionis Pharmaceuticals | Regulatory Ionis well-positioned for continued momentum and substantial value creation in 2026 with two new independent launches and several pivotal data readouts ionis.com ↗
sNDA for sHTG submitted for review |
| 2025-12-01 | Ionis Pharmaceuticals | Regulatory Ionis receives U.S. FDA Breakthrough Therapy designation for olezarsen for severe hypertriglyceridemia (sHTG) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olezarsen as an adjunct to diet to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (sHTG) (TG greater than or equal to 500 mg/dL). |
| 2025-11-08 | Sobi, Inc. | Results Clinically meaningful pivotal study results for olezarsen in sHTG presented as a late breaker at AHA Scientific Sessions sobi.com ↗
The studies met the primary endpoint, with olezarsen achieving a highly statistically significant placebo-adjusted mean reduction in fasting triglyceride (TG) levels of up to 72% at six months. |
| 2025-11-08 | Ionis Pharmaceuticals | Results Groundbreaking pivotal study results of olezarsen for severe hypertriglyceridemia (sHTG) presented as a late breaker at AHA Scientific Sessions ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). |
| 2025-09-19 | Ionis Pharmaceuticals | Regulatory TRYNGOLZA® (olezarsen) approved in the European Union for familial chylomicronemia syndrome (FCS) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that TRYNGOLZA® (olezarsen) has been approved in the European Union (EU) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). |
| 2025-09-02 | Ionis Pharmaceuticals | Results Olezarsen significantly reduces triglycerides and acute pancreatitis events in landmark pivotal studies for people with severe hypertriglyceridemia (sHTG) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). |
| 2025-07-25 | Ionis Pharmaceuticals | Regulatory TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for familial chylomicronemia syndrome (FCS) ionis.com ↗
The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2025. |
| 2025-05-19 | Ionis Pharmaceuticals | Results Ionis announces positive topline results from Essence study of olezarsen in people with moderately elevated triglycerides ionis.com ↗
The trial met its primary endpoint with a statistically significant placebo-adjusted 61% and 58% reduction in triglyceride (TG) levels at 6 months with the 80 mg and 50 mg monthly doses, respectively (p <0.0001). |
| 2024-12-19 | Ionis Pharmaceuticals | Regulatory TRYNGOLZA™ (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet ionis.com ↗ |
| 2024-06-25 | Ionis Pharmaceuticals | Regulatory Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome (FCS). |
| 2024-04-07 | Ionis Pharmaceuticals | Results Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced full results from the Phase 3 Balance study of Ionis' lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). |
| 2024-02-15 | Ionis Pharmaceuticals | Regulatory Olezarsen receives Orphan Drug designation from U.S. FDA for familial chylomicronemia syndrome ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine olezarsen for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP). |
| 2023-09-26 | Ionis Pharmaceuticals | Results Ionis announces positive olezarsen topline results from Phase 3 study in people with familial chylomicronemia syndrome ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results for the Phase 3 Balance study of olezarsen in people with familial chylomicronemia syndrome (FCS). |
| 2023-01-31 | Ionis Pharmaceuticals | Regulatory Ionis receives FDA Fast Track designation for olezarsen in patients with familial chylomicronemia syndrome ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted olezarsen Fast Track designation for the treatment of familial chylomicronemia syndrome (FCS). |
| 2020-08-29 | Ionis Pharmaceuticals | Results Positive Phase 2 Clinical Data of AKCEA-APOCIII-L(Rx) Presented at ESC Congress 2020 ionis.com ↗
Results in late-breaking presentation show patients with hypertriglyceridemia experienced dose-dependent reductions in triglyceride levels, apoC-III, and significant reductions in atherogenic lipoproteins |
| 2020-01-22 | Ionis Pharmaceuticals | Results Akcea and Ionis report positive topline Phase 2 results of AKCEA-APOCIII-L Rx ionis.com ↗ |
| 2017-10-31 | Ionis Pharmaceuticals | Results Akcea and Ionis Report Positive Phase 1/2a Data with AKCEA-APOCIII-LRx ionis.com ↗
today announced positive results of a Phase 1/2a clinical study with AKCEA-APOCIII-LRx. |
All press releases naming this drug 32 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | olezarsen | “This clinical trial evaluated olezarsen, an N-acetyl-galactosamine-conjugated antisense oligonucleotide targeted to hepatic APOC3 mRNA to inhibit apolipoprotein C-III...” PMID 35025993 ↗ Apr 2022 |
| Known as | AKCEA-APOCIII-LRx | “AKCEA-APOCIII-LRx is an N-acetyl galactosamine-conjugated antisense oligonucleotide targeted to the liver that selectively inhibits apoC-III protein synthesis.” PMID 31329855 ↗ Sep 2019 |
| Known as | IONIS- APOCIII-LRx | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650438 ↗ |
| Known as | ISIS 678354 | “Olezarsen (ISIS 678354) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)” NCT06360237 ↗ |
| Known as | ISIS-APOCIII-LRX | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650438 ↗ |
| Action | Inhibit | “selectively inhibits apoC-III protein synthesis” PMID 31329855 ↗ Sep 2019 |
| Modality | Antisense oligonucleotide | “AKCEA-APOCIII-LRx is an N-acetyl galactosamine-conjugated antisense oligonucleotide targeted to the liver that selectively inhibits apoC-III protein synthesis.” PMID 31329855 ↗ Sep 2019 |
| Route | Subcutaneous | “Single-dose cohorts were treated with 10, 30, 60, 90, and 120 mg subcutaneously (sc)” PMID 31329855 ↗ Sep 2019 |
| Target | APOC3 | “N-acetyl galactosamine-conjugated antisense drug to APOC3 mRNA” PMID 31329855 ↗ Sep 2019 |