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Efficacy
Efficacy
Tegsedi significantly delays disease progression vs placebo(1)
Tegsedi significantly delays disease progression vs placebo1
Earlier initiation of Tegsedi demonstrated better outcomes in neuropathic progression2*
Tegsedi significantly stabilise neuropathy-related QoL vs placebo1,2
Tegsedi sustains neuropathy-related QoL stabilisation over 2 years1,2
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References
mNIS+7: modified Neuropathy Impairment Score+7 composite score
*No statistical analysis performed
Tegsedi 284mg is the same as the study drug inotersen sodium 300mg. TTR: transthyretin; OLE: open label extension
- Tegsedi Summary of Product Characteristics. April 2021.
- Benson MD et al. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):22–31.
- Brannagan TH, Wang AK, Coelho T, et al. Eur J Neurol. 2020;27(8):1374-1381. doi:10.1111/ene.14285
- Coelho T et al. Efficacy and safety with >3 years of inotersen treatment for the polyneuropathy of hereditary transthyretin amyloidosis. OPR15, 7th Congress of the European Academy of Neurology 2021, 19-22 June 20.
NEURO-TTR study design2
A phase 3, multi-centre, double-blind, randomised, placebo-controlled study. 15-month treatment period.
Patients
- Adults 18 to 82 years of age
- Stage 1 or stage 2 hATTR polyneuropathy
- Neuropathy Impairment Score ≥10 and ≤130;
Randomly assigned 2:1
- Tegsedi (284 mg weekly subcutaneous dose; n=112)
- Placebo (n=60)
Primary end points
- Change in mNIS+7 (range, -22.3 to 346.3, with higher scores indicating poorer function)
- Change in Norfolk QoL-DN questionnaire score (range, -4 to 136, with higher scores indicating poorer quality of life). A decrease in scores indicated improvement.
NEURO-TTR OLE study design3
Inclusion criteria
Satisfactory completion of treatment in NEURO-TTR.
Patient groups
Patients receiving Tegsedi in NEURO-TTR continued to receive Tegsedi (Tegsedi-Tegsedi group). Patients receiving placebo in NEURO-TTR switched to Tegsedi (placebo-Tegsedi group).
Administration
Patients received 284 mg Tegsedi once weekly via subcutaneous injection for up to 260 weeks (5 years).