Summary

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• Tegsedi (inotersen) is indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Inotersen significantly reduced the progression of peripheral neuropathy, as indicated by a decrease of -19.73 points (95% CI -26.50 to -12.96; P < 0.001) in the modified Neuropathy Impairment Score plus 7 nerve tests (NIS+7) at week 66, and improved quality of life by -10.85 points (95% CI -17.25 to -4.45; P < 0.001) in the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) total score at week 65.
• Comparative treatments, such as patisiran, demonstrated a greater reduction in peripheral neuropathy (modified NIS+7 by -33.99 points; P < 0.001) and an improvement in quality of life (Norfolk QOL-DN by -21.10 points; P < 0.001) relative to placebo, while diflunisal and tafamidis also showed some benefits in reducing neuropathy progression, though with varying degrees of effectiveness.
• No specific differences in effectiveness among different population types or subgroups were reported for inotersen.
• Inotersen may slightly increase mortality (RR 5.94; 95% CI 0.33 to 105.60; P = 0.22; low-certainty evidence) and severe adverse events (RR 1.48; 95% CI 0.85 to 2.57; P = 0.16; low-certainty evidence) compared to placebo. Additionally, there were more dropouts due to adverse events in the inotersen group compared to placebo (RR 8.57; 95% CI 1.16 to 63.07; P = 0.035; low-certainty evidence).
• Tafamidis, diflunisal, and patisiran showed no clear differences in mortality, dropouts due to adverse events, or severe adverse events compared to placebo, with varying levels of certainty evidence ranging from very low to low.
• The studies primarily focus on adults with familial amyloid polyneuropathy (FAP) and transthyretin amyloidosis (ATTR-PN). In early-stage TTR-FAP, tafamidis showed a slight reduction in peripheral neuropathy progression. Tafamidis also significantly improved all-cause mortality and cardiovascular hospitalizations in both variant and wild-type transthyretin cardiac amyloidosis (CA).