TEGSEDI

Tegsedi is the brand name for inotersen, an antisense oligonucleotide therapy used to treat adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR‑PN). It works by reducing the liver’s production of transthyretin (TTR) protein, which forms amyloid deposits in nerves and other tissues.

Mechanism and effects: Inotersen binds to TTR messenger RNA and promotes its degradation, lowering circulating TTR levels. By decreasing the amount of TTR available to form amyloid, Tegsedi aims to slow disease progression and may help stabilize or improve neuropathic symptoms in eligible patients.

Indication and administration: Tegsedi is indicated for adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy. It is administered as a subcutaneous injection of 284 mg once weekly. The injections can be given at home by patients or trained caregivers, after appropriate instruction and monitoring.

Safety and monitoring: Tegsedi carries a risk of serious adverse effects, notably thrombocytopenia and glomerulonephritis. Because of these risks, it is available under a risk evaluation and mitigation program (REMS) that requires regular monitoring of platelet counts and renal function before and during treatment. Common adverse events include injection-site reactions, headache, fatigue, nausea, and fever. Dose interruptions or discontinuation may be necessary if safety thresholds are not met.

Regulatory status and context: Tegsedi was approved for adult patients with hATTR‑PN in the United States in 2018. It is part of a broader category of TTR-lowering therapies for this condition, alongside other approaches such as RNA interference–based treatments.