Drug updated on 5/17/2024
Dosage Form | Injection (intraveneous; 10 mg/5 mL [2 mg/mL]) |
Drug Class | Transthyretin-directed small interfering RNA |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Latest News
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Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Onpattro (patisiran) Prescribing Information. | 2023 | Alnylam Pharmaceuticals Inc., Cambridge, MA |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Prevalence and clinical outcomes of transthyretin amyloidosis: a systematic review and meta-analysis. | 2022 | European Journal of Heart Failure |
The impact of clinical heterogeneity on conducting network meta-analyses in transthyretin amyloidosis with polyneuropathy. | 2020 | Current Medical Research and Opinion |
Pharmacological treatment for familial amyloid polyneuropathy. | 2020 | The Cochrane Database of Systematic Reviews |
RNA drugs and RNA targets for small molecules: principles, progress, and challenges. | 2020 | Pharmacological Reviews |
Overview of treatments used in transthyretin-related hereditary amyloidosis: a systematic review. | 2020 | European Journal of Hospital Pharmacy |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Diagnosis and screening of patients with hereditary transthyretin amyloidosis (hATTR): current strategies and guidelines. | 2020 | Therapeutics and Clinical Risk Management |
Leading RNA interference therapeutics part 1: silencing hereditary transthyretin amyloidosis, with a focus on patisiran. | 2020 | Molecular Diagnosis and Therapy |