Drug updated on 4/18/2024
| Dosage Form | Injection (subcutaneous; 25 mg/0.5 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.
Summary
- Vutrisiran (Amvuttra) is indicated for the treatment of polyneuropathy in adults suffering from hereditary transthyretin-mediated amyloidosis, a rapidly progressing and potentially fatal condition if left untreated.
- One study was reviewed to gather information about this drug's effectiveness and safety profile compared to tafamidis, another approved treatment for the same condition.
- The comparison between Vutrisiran (Amvuttra) and tafamidis showed that Vutrisiran had a greater effect on improving symptoms such as neuropathy impairment score-lower limbs (NIS-LL), with a statistically significant relative mean change of -5.3 (95% CI: -9.4, -1.2; p = 0.011).
- In terms of quality of life measured by the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) score, Vutrisiran also demonstrated superior improvement with a relative mean change in Norfolk QOL-DN score of -18.3 (95% CI: -28.6, -8.0; p < 0.001).
- Furthermore, when considering nutritional status reflected by the Modified Body Mass Index (mBMI), which is crucial for patients with this condition, Vutrisiran again outperformed tafamidis, showing an improved mBMI value of 63 (95% CI: 10, 117; p=0.20).
- The analysis suggests that Vutrisiran may offer substantial benefits due to its broad efficacy across specific subgroups within the adult population. However, the study does not provide detailed safety data comparing both drugs, hence it's important to consider broader profiles (efficacy, safety, patient characteristics) when evaluating Vutrisiran's use.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Amvuttra (vutrisiran) Prescribing Information. | 2022 | Alnylam Pharmaceuticals Inc., Cambridge, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy | 2023 | Expert Opinion on Pharmacotherapy |