Drug updated on 3/28/2024
| 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
- To treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Summary
- Patisiran (Onpattro) is used to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
- A total of 4 systematic reviews/meta-analyses were reviewed for information on patisiran's effectiveness and safety profile.
- In a study involving various patient subgroups, it was found that 2-year survival rates were higher among patients treated with tafamidis/patisiran compared to those following a natural disease course (79.9% vs 72.4%) indicating its potential therapeutic benefit.
- Another review highlighted that patisiran significantly reduces plasma TTR levels and improves neuropathy and quality of life compared to placebo, demonstrating its efficacy in managing symptoms associated with hereditary peripheral neuropathies.
- However, one analysis noted conflicting results from different studies regarding the impact of patisiran on cardiac imaging parameters suggesting further research may be needed for conclusive evidence about this aspect of treatment outcomes.
- Despite promising results shown by subgroup analysis in variant transthyretin-CA patients treated with patisiran, these findings need confirmation through randomized controlled trials as suggested by another systematic review included in this dataset.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Onpattro (patisiran) Prescribing Information. | 2022 | 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 |
| Targeting transthyretin - Mechanism-based treatment approaches and future perspectives in hereditary amyloidosis. | 2021 | Journal of Neurochemistry |
| Targeted therapies for hereditary peripheral neuropathies: systematic review and steps towards a ‘treatabolome’. | 2021 | Journal of Neuromuscular Diseases |
| Clinical review report: Patisiran (Onpattro). | 2019 | CADTH |
| Specific therapy for transthyretin cardiac amyloidosis: a systematic literature review and evidence‐based recommendations. | 2019 | Journal of the American Heart Association |
| Inotersen and patisiran for hereditary transthyretin amyloidosis: effectiveness and value. | 2018 | ICER |
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 |