Summary

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• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• Cardiac Effectiveness Outcomes: Vutrisiran significantly reduced N-terminal pro B-type natriuretic peptide (NT-proBNP) levels in the modified intention to treat (mITT) population (adjusted geometric mean fold change ratio: 0.480, 95% confidence interval (CI): 0.383-0.600, p = 9.606 x 10^(-10)) and the cardiac subpopulation (0.491, 95% CI: 0.337-0.716, p = 0.0004). Trends towards improvement in echocardiographic parameters were observed, with 68.1% showing reduced left ventricular total uptake and 64.6% reduced heart-to-contralateral lung ratio on (99m)Tc scintigraphy.
• Neuropathy and Quality of Life Outcomes: Vutrisiran met the primary endpoint by improving modified neuropathy impairment score (mNIS)+7 score at 9 months (p = 3.54 x 10^(-12)) and demonstrated additional improvements in secondary endpoints, including the Norfolk Quality of Life-Diabetic Neuropathy and 10-meter walk test at both 9 and 18 months.
• Comparison with Patisiran: Vutrisiran was non-inferior to patisiran in reducing transthyretin (TTR) levels, with both drugs showing improvements in multiple disease-relevant outcomes.
• Most adverse events associated with vutrisiran were mild or moderate in severity, with no drug-related discontinuations or deaths reported.
• No increase in cardiac adverse events was observed in the vutrisiran group compared to the external placebo.
• There is no population types or subgroups information available in the reviewed documents.