Is Tegsedi an Injectable for hATTR Amyloidosis?
Yes, Tegsedi (inotersen) is an injectable treatment for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.[1]
How Is Tegsedi Administered?
Patients self-administer Tegsedi via subcutaneous injection once weekly, typically 284 mg (0.75 mL) using a prefilled syringe. It's given in the abdomen, upper arm, or thigh, with site rotation to avoid irritation.[1][2]
What Does Tegsedi Treat Specifically?
Tegsedi targets hATTR amyloidosis with polyneuropathy, a rare disease where mutated transthyretin protein forms amyloid deposits damaging nerves. It reduces transthyretin production in the liver via antisense oligonucleotide mechanism, slowing neuropathy progression.[1]
Common Side Effects Patients Report
Injection site reactions (redness, pain) occur in most patients. Other issues include thrombocytopenia (low platelets, requiring monitoring), vitamin A deficiency, and renal effects. FDA black box warns of severe thrombocytopenia and bleeding risks.[1][2]
How Does Tegsedi Compare to Other hATTR Treatments?
| Treatment | Type | Mechanism | Key Difference |
|-----------|------|-----------|---------------|
| Tegsedi (inotersen) | Weekly SC injection | Antisense oligonucleotide (TTR reduction) | Broad TTR lowering; high thrombocytopenia risk |
| Onpattro (patisiran) | IV infusion every 3 weeks | siRNA (TTR silencing) | Lipid nanoparticle delivery; less platelet impact |
| Vutrisiran (Amvuttra) | SC every 3 months | siRNA (TTR silencing) | Longer dosing interval; similar efficacy to patisiran |
| Tafamidis (Vyndamax/Vyndaqel) | Oral daily | TTR stabilizer | Prevents misfolding; no injection needed, broader hATTR use |
Tegsedi showed neuropathy improvement in NEURO-TTR trial vs. placebo.[1][3]
Who Makes Tegsedi and What's the Status?
Ionis Pharmaceuticals developed it; Akcea Therapeutics (now part of Ionis) markets it. FDA approved 2018 in US, EMA 2019 in EU. Available via restricted programs due to safety monitoring.[1]
Patent and Pricing Details
Key US patents on inotersen composition expire around 2031-2034; check DrugPatentWatch.com for challenges or extensions.[4] List price ~$450,000/year US; patient assistance programs reduce costs for eligible.[2]
[1]: FDA Tegsedi Label - https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/211992s000lbl.pdf
[2]: Tegsedi HCP Site - https://www.tegsedi.com/
[3]: NEJM Study (NEURO-TTR) - https://www.nejm.org/doi/full/10.1056/NEJMoa1802266
[4]: DrugPatentWatch.com - Tegsedi Patents - https://www.drugpatentwatch.com/p/tradename/TEGSEDI