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) | |
| |
|
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 U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9]
Inotersen was approved for medical use in the European Union in July 2018. [5]
The U.S. 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 patients 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 patients with hereditary transthyretin-mediated amyloidosis. [6] Patients 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, patients received three doses of treatment, followed by once weekly subcutaneous injections for 64 weeks. [6] Neither the patients nor the health care providers knew which treatment was being given until after the trial was completed. [6]
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) | |
| |
|
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 U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9]
Inotersen was approved for medical use in the European Union in July 2018. [5]
The U.S. 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 patients 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 patients with hereditary transthyretin-mediated amyloidosis. [6] Patients 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, patients received three doses of treatment, followed by once weekly subcutaneous injections for 64 weeks. [6] Neither the patients nor the health care providers knew which treatment was being given until after the trial was completed. [6]