Drugs / Inotersen
Inotersen
also known as
GSK 299872 · GSK-2998728 · INOTERSEN SODIUM · Isis 420915 · ISIS 420915 SODIUM · ISIS-420915 SODIUM SALT · TEGSEDI
Antisense oligonucleotide targets TTR via inhibition
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hereditary amyloidosis 2 milestones | |||||
| Filed | US (FDA) | — | hereditary amyloidosis | 2017-11-06 | “submitted a new drug application (NDA) to the U.S. Food and Drug Administration for inotersen, an investigational medicine for the treatment of patients with hereditary TTR...” ionis.com ↗ |
| Filed | EU (EMA) | — | hereditary amyloidosis | 2017-11-03 | “submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for inotersen” ionis.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | US (FDA) | TEGSEDI | — | 2018-10-05 | fda.gov ↗ |
| Approved | EU (EMA) | Tegsedi | — | 2018-07-06 | europa.eu ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 22 trials | ||||||
| Phase 2 | NCT03702829 | Feb 2019 → Dec 2021 | amyloidosis | Brigham and Women's Hospital | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT02627820 | Jan 2016 → Dec 2018 | amyloidosis | Brigham and Women's Hospital | Withdrawn | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT02175004 | Jun 2014 → Sep 2020 | classic or attenuated familial adenomatous polyposis, familial amyloid neuropathy | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT04306510 | Jan 2021 → Mar 2024 | familial amyloid neuropathy | Akcea Therapeutics | Terminated | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT03400098 | — | hereditary amyloidosis | Ionis Pharmaceuticals, Inc. | Approved for marketing | No outcome recorded |
| Comparator or background therapy2 trials | ||||||
| Phase 2/3 | NCT01737398 Comparator | Mar 2013 → Mar 2017 | classic or attenuated familial adenomatous polyposis, familial amyloid neuropathy | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT04136184 Comparator | Dec 2019 → Apr 2023 | familial amyloid neuropathy | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
News releases announcing trial results or a regulatory action · 20
| Date | Issuer | Release |
|---|---|---|
| 2021-10-11 | PTC Therapeutics | Regulatory Tegsedi® Receives Innovative Drug Category Pricing in Brazil ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Tegsedi® (inotersen) has successfully received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos - CMED (Drug Market Regulation Chamber) in Brazil. |
| 2020-05-28 | Ionis Pharmaceuticals | Results Akcea and Ionis Announce Publication of Long-Term Clinical Data of TEGSEDI® in Patients with Polyneuropathy Driven by Hereditary Transthyretin Amyloidosis Demonstrating Sustained Improvements and Even Greater Stabilization in Patients Starting Earlier Treatment ionis.com ↗
This interim analysis, published in the European Journal of Neurology, show that treatment with TEGSEDI was not associated with additional safety concerns or signs of increased toxicity in study participants treated for up to five years. |
| 2019-10-29 | PTC Therapeutics | Regulatory PTC Therapeutics Reports Third Quarter 2019 Financial Results and Provides a Corporate Update ptcbio.com ↗
Tegsedi® (inotersen) received approval from the Brazilian health regulatory authority (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR), to delay disease progression and improve quality of life. |
| 2019-05-07 | Ionis Pharmaceuticals | Results New long-term TEGSEDI safety and efficacy data featured as an oral presentation at 2019 American Academy of Neurology Annual Meeting (AAN) ionis.com ↗
NEURO-TTR phase 3 open-label extension study showed TEGSEDI® (inotersen) treatment continued to slow progression over two-year period in patients with Hereditary ATTR amyloidosis with polyneuropathy |
| 2019-03-15 | Ionis Pharmaceuticals | Results Akcea and Ionis announce upcoming data presentations at the 2019 American College of Cardiology Meeting (ACC.19) ionis.com ↗
Presentations focus on the benefits of treatment with TEGSEDI™ (inotersen) and results show continued efficacy with long-term exposure in the NEURO-TTR open-label extension study |
| 2018-10-05 | Ionis Pharmaceuticals | Regulatory Akcea Announces Its Access and Distribution Strategy for TEGSEDI™ (inotersen) ionis.com ↗
Approved by the U.S. Food and Drug Administration (FDA) today, TEGSEDI is the first and only subcutaneous RNA-targeting therapeutic offering patients an effective treatment for the polyneuropathy of hATTR amyloidosis. |
| 2018-10-05 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Receive FDA Approval of TEGSEDI™ (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults ionis.com ↗ |
| 2018-10-04 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Announce Approval of TEGSEDI™ (inotersen injection) in Canada ionis.com ↗ |
| 2018-07-11 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Announce Approval of TEGSEDI™ (inotersen) in the European Union ionis.com ↗ |
| 2018-07-05 | Ionis Pharmaceuticals | Results New England Journal of Medicine Publishes Results from Pivotal Study of TEGSEDI™ (inotersen) for the Treatment of Hereditary ATTR Amyloidosis ionis.com ↗ |
| 2018-06-01 | Ionis Pharmaceuticals | Regulatory Akcea and Ionis Receive Positive EU CHMP Opinion for TEGSEDI ionis.com ↗ |
| 2018-03-26 | Ionis Pharmaceuticals | Results Ionis and Akcea Present New Data from ATTR Amyloidosis Program at 16th International Symposium on Amyloidosis ionis.com ↗
Continued benefit with long-term inotersen treatment observed in both quality of life (Norfolk-QOL-DN) and neuropathy (mNIS+7) measures in hATTR amyloidosis patients; |
| 2018-01-08 | Ionis Pharmaceuticals | Regulatory Ionis' Inotersen NDA Accepted for Priority Review by the FDA ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that its New Drug Application (NDA) for inotersen has been accepted for Priority Review by the U.S. Food and Drug Administration (FDA). |
| 2017-11-06 | Ionis Pharmaceuticals | Regulatory Ionis Announces Submission of New Drug Application (NDA) for Inotersen to the U.S. FDA ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the company submitted a new drug application (NDA) to the U.S. Food and Drug Administration for inotersen, an investigational medicine for the treatment of patients with hereditary TTR amyloidosis (hATTR). |
| 2017-11-03 | Ionis Pharmaceuticals | Regulatory Ionis Announces Submission of Marketing Authorization Application for Inotersen to the European Medicines Agency ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the company submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for inotersen, based on the phase 3 NEURO-TTR study in patients with hereditary TTR amyloidosis (hATTR). |
| 2017-11-02 | Ionis Pharmaceuticals | Results Ionis Presents New Data from NEURO-TTR Study at European ATTR Amyloidosis Meeting ionis.com ↗
Results from the study demonstrated early and significant benefit in both co-primary endpoints: Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, in inotersen-treated patients compared to placebo-treated. |
| 2017-10-15 | Ionis Pharmaceuticals | Results Ionis Presents New Data from NEURO-TTR Study and Highlights Programs from Its Neurological Disease Franchise at ANA Congress ionis.com ↗
In the study, inotersen-treated patients achieved a mean 19.73-point benefit in the mNIS+7 co-primary endpoint after 15 months of treatment, compared to placebo-treated patients (p = 0.00000004), further demonstrating the clinically meaningful benefit of inotersen treatment. |
| 2017-08-11 | Ionis Pharmaceuticals | Regulatory Ionis to Independently Advance Inotersen and IONIS-FB-L Rx ionis.com ↗
Ionis plans to file for marketing authorization for inotersen this year to support a commercial launch of inotersen in 2018. |
| 2017-07-10 | Ionis Pharmaceuticals | Results New Data Presented at Peripheral Nerve Society Meeting Further Support Potential Benefit of Inotersen ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) presented new top-line data from the Phase 3 NEURO-TTR study of inotersen in patients with familial amyloid polyneuropathy (FAP) at the 2017 Peripheral Nerve Society Meeting. |
| 2017-05-15 | Ionis Pharmaceuticals | Results Ionis Pharmaceuticals Announces Phase 3 NEURO-TTR Study of Inotersen (IONIS-TTR Rx) Meets Both Primary Endpoints ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the Phase 3 NEURO-TTR study of inotersen (IONIS-TTRRx) in patients with familial amyloid polyneuropathy (FAP) met both primary endpoints. |
All press releases naming this drug 26 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Inotersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04136184 ↗ |
| Known as | GSK 299872 | “Isis 420915/GSK 299872, an antisense oligonucleotide.” NCT02627820 ↗ |
| Known as | GSK-2998728 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297770 ↗ |
| Known as | INOTERSEN SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297770 ↗ |
| Known as | Isis 420915 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02627820 ↗ |
| Known as | ISIS 420915 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297770 ↗ |
| Known as | ISIS-420915 SODIUM SALT | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297770 ↗ |
| Known as | TEGSEDI | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04306510 ↗ |
| Action | Inhibit | “Inotersen, a 2'- O-methoxyethyl-modified antisense oligonucleotide, inhibits hepatic production of transthyretin.” PMID 29972757 ↗ Jul 2018 |
| Modality | Antisense oligonucleotide | “Inotersen (also known as ISIS 420915) is an antisense drug” NCT01737398 ↗ |
| Route | Subcutaneous | “300 mg inotersen administered subcutaneously (SC)” NCT01737398 ↗ |
| Target | TTR | “decrease the amount of mutant and normal TTR made by the liver” NCT01737398 ↗ |