Drug updated on 4/18/2024
| Dosage Form | Capsule (oral; 80 mg) |
| Drug Class | Transthyretin stabilizers |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.
Summary
- Tafamidis meglumine (Vyndaqel) is indicated for the treatment of cardiomyopathy in adults with wild type or hereditary transthyretin-mediated amyloidosis, reducing cardiovascular mortality and hospitalization.
- Eight studies have been reviewed to gather information about this drug's efficacy and safety profile.
- The studies consistently reported tafamidis as efficacious and safe for patients with transthyretin amyloid cardiomyopathy, significantly reducing cardiovascular mortality and all-cause death. However, there was no direct comparison made between tafamidis' effectiveness or safety compared to other drugs within its therapeutic class.
- Subgroup analysis revealed that the efficacy of tafamidis does not differ significantly between patients with wild-type or hereditary ATTR. However, it was noted that genotypic variability among hereditary ATTR might influence treatment outcomes.
- Despite positive reports on its impact on mortality rates and hospitalizations due to cardiovascular issues, more randomized controlled trials are needed to validate certain clinical parameters associated with tafamidis use.
- While indirect comparisons suggest a differentiated landscape of ATTR treatments where Vyndaqel plays a crucial role in managing cardiomyopathy aspects; additional research is required for direct comparisons against other therapies like patisiran and inotersen which focus primarily on peripheral neuropathy aspects of ATTR.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Vyndaqel (tafamidis meglumine) Prescribing Information. | 2023 | Pfizer Inc., New York, NY |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| JCS 2020 Guideline on diagnosis and treatment of cardiac amyloidosis. | 2020 | Circulation Journal |
| CCS/CHFS heart failure guidelines: clinical trial update on functional mitral regurgitation, SGLT2 inhibitors, ARNI in HFpEF, and tafamidis in amyloidosis. | 2020 | Canadian Journal of Cardiology |
| Canadian Cardiovascular Society/Canadian Heart Failure Society joint position statement on the evaluation and management of patients with cardiac amyloidosis. | 2020 | Canadian Cardiovascular Society |
| Cardiac amyloidosis: evolving diagnosis and management. | 2020 | American Heart Association |