Biology:Inotersen
| Clinical data | |
|---|---|
| Trade names | Tegsedi |
| Other names | GSK-2998728, ISIS-420915 |
| AHFS/Drugs.com | Monograph |
| License data | |
| Routes of administration | Subcutaneous |
| Drug class | Antisense oligonucleotides |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| PubChem CID | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C230H318N69O121P19S19 |
| Molar mass | 7183.08 g·mol−1 |
| 3D model (JSmol) | |
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Inotersen, sold under the brand name Tegsedi, is a 2'-O-(2-methoxyethyl) (2'-MOE) antisense oligonucleotide medication used for the treatment of nerve damage in adults with hereditary transthyretin-mediated amyloidosis.[5][6] The sequence is TCTTG GTTACATGAA ATCCC, where C is methylated C, and the first and third section (bases 1-5 and 16–20, separated from the middle section by spaces) are MOE-modified.[7]
The most common side effects are injection site reactions (redness, swelling, bleeding, pain, rash, and itching at the injection site), nausea, headache, tiredness, low platelet counts, and fever.[5]
Inotersen can cause serious side effects, including low platelet counts and kidney inflammation.[5] Because of these serious side effects, Inotersen is available in the United States only through a restricted program called the Tegsedi Risk Evaluation and Mitigation (REMS) Program.[5]
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[8]
History
Inotersen was approved for medical use in the European Union in July 2018.[4]
The U.S. Food and Drug Administration (FDA) approved inotersen in October 2018.[5] The application for inotersen was granted orphan drug designation.[9]
The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 patients with hereditary transthyretin-mediated amyloidosis.[5] The trial was conducted at 24 sites in Australia, Europe, South America, and the United States.[5]
The benefits and side effects of inotersen were evaluated in one clinical trial that enrolled patients with hereditary transthyretin-mediated amyloidosis.[5] Patients were randomly assigned to receive inotersen or placebo by subcutaneous injection given once a week for 65 weeks.[5] During the first week of treatment, patients received three doses of treatment, followed by once weekly subcutaneous injections for 64 weeks.[5] Neither the patients nor the health care providers knew which treatment was being given until after the trial was completed.[5]
References
- ↑ "PrTEGSEDITM inotersen injection". Product Monograph Including Patient Medication Information. Akcea Therapeutics, Inc.. https://pdf.hres.ca/dpd_pm/00064890.PDF.
- ↑ "Tegsedi 284 mg solution for injection in pre-filled syringe - Summary of Product Characteristics (SmPC)". 15 October 2019. https://www.medicines.org.uk/emc/product/10011.
- ↑ "Tegsedi- inotersen injection, solution". 2 September 2020. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543.
- ↑ 4.0 4.1 "Tegsedi EPAR". 17 September 2018. https://www.ema.europa.eu/en/medicines/human/EPAR/tegsedi.
- ↑ 5.00 5.01 5.02 5.03 5.04 5.05 5.06 5.07 5.08 5.09 5.10 "Drug Trial Snapshot: Tegsedi". 23 July 2019. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trial-snapshot-tegsedi.
This article incorporates text from this source, which is in the public domain.
- ↑ "Inotersen (transthyretin-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis". Neurodegenerative Disease Management 9 (1): 25–30. February 2019. doi:10.2217/nmt-2018-0037. PMID 30561247.
- ↑ "Population Pharmacokinetic-Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis". Nucleic Acid Therapeutics 30 (3): 153–163. June 2020. doi:10.1089/nat.2019.0822. PMID 32286934.
- ↑ (PDF) New Drug Therapy Approvals 2018 (Report). January 2019. https://www.fda.gov/media/120357/download. Retrieved 16 September 2020.
- ↑ "Inotersen Orphan Drug Designation and Approval". 24 July 2012. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=374512. This article incorporates text from this source, which is in the public domain.
External links
- "Inotersen". Drug Information Portal. U.S. National Library of Medicine (NLM). https://druginfo.nlm.nih.gov/drugportal/name/inotersen.
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