Inotersen, sold under the brand name Tegsedi, is a 2'-O-(2-methoxyethyl) (2'-MOE)

antisense oligonucleotide Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small fragments of nucleic aci ...

medication used for the treatment of nerve damage in adults with

hereditary transthyretin-mediated amyloidosis Familial amyloid polyneuropathy, also called hereditary transthyretin amyloidosis (hATTR), or Corino de Andrade's disease, is an autosomal dominant neurodegenerative disease. It is a form of amyloidosis, and was first identified and described by ...

. 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. 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. Inotersen can cause serious side effects, including low platelet counts and kidney inflammation. 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. The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) considers it to be a

first-in-class medication A first-in-class medication is a prototype drug that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medic ...

History

Inotersen was approved for medical use in the European Union in July 2018. The US

(FDA) approved inotersen in October 2018. The application for inotersen was granted

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designation. The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 participants with hereditary transthyretin-mediated amyloidosis. The trial was conducted at 24 sites in Australia, Europe, South America, and the United States. The benefits and side effects of inotersen were evaluated in one clinical trial that enrolled participants with hereditary transthyretin-mediated amyloidosis. Participants were randomly assigned to receive inotersen or placebo by subcutaneous injection given once a week for 65 weeks. During the first week of treatment, participants received three doses of treatment, followed by once weekly subcutaneous injections for 64 weeks. Neither the participants nor the health care providers knew which treatment was being given until after the trial was completed.

References

{{Authority control Antisense RNA Orphan drugs