Inotersen (Tegsedi) is a prescription medicine used to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is an antisense oligonucleotide, which works by decreasing the production of a certain protein that can cause amyloid deposits to form in the body. Inotersen is given as an injection under the skin (subcutaneous injection) once a week. It may help improve nerve function and slow the progression of the disease.
Inotersen is a medication specifically designed to address a rare genetic condition called hereditary transthyretin amyloidosis (hATTR). Here's a breakdown of what you should know:
What is hATTR?
- hATTR is an inherited disease where a mutation in the transthyretin (TTR) gene leads to the production of abnormal TTR protein.
- This abnormal protein misfolds and accumulates as amyloid deposits in various organs and tissues, causing progressive damage.
- Symptoms of hATTR can include nerve damage (polyneuropathy), heart problems, and eye issues.
How Inotersen Works:
- Inotersen belongs to a class of drugs called antisense oligonucleotides (ASOs).
- It targets the TTR messenger RNA (mRNA), the genetic blueprint for TTR protein production, inside liver cells.
- By binding to the TTR mRNA, inotersen triggers its degradation, thereby reducing the production of both mutant and healthy TTR protein.
- This decrease in TTR protein levels can slow down the progression of hATTR and potentially improve symptoms.
Applications:
- Inotersen is approved by the FDA to treat the polyneuropathy (nerve damage) associated with hATTR in adults.
Dosage and Administration:
- Inotersen is administered as a subcutaneous injection (under the skin) typically once weekly by a healthcare professional or trained caregiver.
Important Considerations:
- Inotersen is a powerful medication with potential side effects, including injection site reactions, kidney problems, low blood cell counts, and nervous system effects.
- Regular monitoring by a doctor is crucial while on inotersen treatment.
- Inotersen is not a cure for hATTR, but it can be a valuable tool in managing the disease and potentially improving quality of life.
Brand Name:
- Inotersen is marketed under the brand name Tegsedi.