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Drug updated on 5/17/2024

Dosage FormInjection (intraveneous; 10 mg/5 mL [2 mg/mL])
Drug ClassTransthyretin-directed small interfering RNA
Ongoing and
Completed Studies


  • Indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

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  • Patisiran (Onpattro) is indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. It has demonstrated effectiveness in reducing disability progression and improving quality of life, with a favorable safety profile indicated by low rates of severe adverse events.
  • The information was derived from five systematic reviews/meta-analyses that focused on various aspects related to therapies for transthyretin-mediated amyloidosis (ATTR), including comparisons between patisiran and other medications such as tafamidis, inotersen, and diflunisal.
  • Survival rates for ATTR patients were found to be improved when treated with patisiran or tafamidis compared to the natural disease course. This suggests that these medications have a positive effect on survival rates among this patient population.
  • A direct comparison between the efficacy and safety profiles of patisiran versus other treatments proved challenging due to heterogeneity in trial designs and population characteristics across different studies.
  • Despite limitations regarding direct comparisons due to sample size constraints, one study provided evidence suggesting all four drugs - patisiran, diflunisal, tafamidis, and inotersen - may benefit TTR-FAP patients; however, it highlighted moderate-to-high certainty specifically about patisiran's effectiveness based on its significant reduction in disability progression compared to placebo.
  • Subgroup considerations are important when tailoring treatment approaches: individual patient characteristics like different ATTR genotypes or stages of disease progression can influence outcomes. Further research is needed for definitive head-to-head comparisons concerning safety and efficacy among available treatments.