Inotersen
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Clinical data | |
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Trade names | Tegsedi |
Other names | GSK-2998728, ISIS-420915 |
AHFS/Drugs.com | Monograph |
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Routes of administration | Subcutaneous |
Drug class | Antisense oligonucleotides |
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Chemical and physical data | |
Formula | C230H318N69O121P19S19 |
Molar mass | 7183.08 g·mol−1 |
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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.[6][7] 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.[8]
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.[6]
Inotersen can cause serious side effects, including low platelet counts and kidney inflammation.[6] 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.[6]
The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[9]
History
[edit]Inotersen was approved for medical use in the European Union in July 2018.[5]
The US Food and Drug Administration (FDA) approved inotersen in October 2018.[6] The application for inotersen was granted orphan drug designation.[10]
The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 participants with hereditary transthyretin-mediated amyloidosis.[6] The trial was conducted at 24 sites in Australia, Europe, South America, and the United States.[6] The benefits and side effects of inotersen were evaluated in one clinical trial that enrolled participants with hereditary transthyretin-mediated amyloidosis.[6] Participants were randomly assigned to receive inotersen or placebo by subcutaneous injection given once a week for 65 weeks.[6] During the first week of treatment, participants received three doses of treatment, followed by once weekly subcutaneous injections for 64 weeks.[6] Neither the participants nor the health care providers knew which treatment was being given until after the trial was completed.[6]
References
[edit]- ^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
- ^ "Tegsedi inotersen injection" (PDF). Product Monograph Including Patient Medication Information. Akcea Therapeutics, Inc. Archived (PDF) from the original on 1 June 2022. Retrieved 1 June 2022 – via The Drug and Health Product Register, Government of Canada.
- ^ "Tegsedi 284 mg solution for injection in pre-filled syringe - Summary of Product Characteristics (SmPC)". (emc). 15 October 2019. Archived from the original on 7 October 2020. Retrieved 3 October 2020.
- ^ "Tegsedi- inotersen injection, solution". DailyMed. 2 September 2020. Archived from the original on 7 October 2020. Retrieved 3 October 2020.
- ^ a b "Tegsedi EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 8 October 2020. Retrieved 3 October 2020.
- ^ a b c d e f g h i j k This article incorporates text from this source, which is in the public domain.
- ^ Benson MD, Dasgupta NR, Monia BP (February 2019). "Inotersen (transthyretin-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis". Neurodegenerative Disease Management. 9 (1): 25–30. doi:10.2217/nmt-2018-0037. PMID 30561247. S2CID 56178847.
- ^ Yu RZ, Collins JW, Hall S, Ackermann EJ, Geary RS, Monia BP, et al. (June 2020). "Population Pharmacokinetic-Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis". Nucleic Acid Therapeutics. 30 (3): 153–163. doi:10.1089/nat.2019.0822. PMC 7249474. PMID 32286934.
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: CS1 maint: overridden setting (link) - ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original on 17 September 2020. Retrieved 16 September 2020.
- ^ This article incorporates text from this source, which is in the public domain.