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Inotersen
Pharmaceutical drug From Wikipedia, the free encyclopedia
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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 US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[8]
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History
Inotersen was approved for medical use in the European Union in July 2018.[4]
The US 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 participants 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 participants with hereditary transthyretin-mediated amyloidosis.[5] Participants 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, participants received three doses of treatment, followed by once weekly subcutaneous injections for 64 weeks.[5] Neither the participants nor the health care providers knew which treatment was being given until after the trial was completed.[5]
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References
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