Amvuttra(vutrisiran)
Neurology· 1525 cited findings· 3 source documents
Company: Alnylam Pharmaceuticals, Inc. →
What the regulatory reviews say
Amvuttra (vutrisiran) is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. It also has an extended indication for the treatment of wild-type or hereditary transthyretin-mediated amyloidosis in adult patients with cardiomyopathy (ATTR-CM). The drug is administered once every 3 months (q3M) at a clinical dose of 25 mg.
Key efficacy findings at Month 18 for EU regulatory submission include a NIS LS mean difference (vutrisiran – placebo) of -22.75, an mBMI LS mean difference (vutrisiran – placebo) of 140.7 kg/m2 × albumin g/L, and an R-ODS LS mean difference (vutrisiran – placebo) of 8.4 points. The NT-proBNP adjusted geometric fold change ratio (vutrisiran/placebo) was 0.480. For safety, 322 patients receiving vutrisiran experienced at least 1 adverse event (AE), compared to 323 patients in the placebo group. Severe AEs were reported in 158 patients on vutrisiran and 194 patients on placebo. Serious adverse events (SAEs) occurred in 201 patients receiving vutrisiran, with 2 SAEs considered related to the study drug.
Amvuttra received orphan medicinal product designation on 25 May 2018. The Marketing Authorisation Application was submitted on 10 September 2021, and a CHMP positive opinion was issued on 21 July 2022.
Key figures — cited to the source page
| NIS LS mean difference (vutrisiran – placebo) | -22.75 | Alnylam B V — Amvuttra EMA EPAR public assessment report (EM, p.77 |
| mBMI LS mean difference (vutrisiran – placebo) | 140.7 kg/m2 × albumin g/L | Alnylam B V — Amvuttra EMA EPAR public assessment report (EM, p.72 |
| R-ODS LS mean difference (vutrisiran – placebo) | 8.4 points | Alnylam B V — Amvuttra EMA EPAR public assessment report (EM, p.73 |
| NT-proBNP adjusted geometric fold change ratio (vutrisiran/placebo) | 0.480 | Alnylam B V — Amvuttra EMA EPAR public assessment report (EM, p.75 |
| Patients with at least 1 AE (vutrisiran) | 322 patients | Alnylam B V — Amvuttra EMA EPAR variation assessment report, p.95 |
| Severe AEs (vutrisiran) | 158 patients | Alnylam B V — Amvuttra EMA EPAR variation assessment report, p.95 |
| SAEs considered related to study drug (vutrisiran) | 2 patients | Alnylam B V — Amvuttra EMA EPAR variation assessment report, p.95 |
| CHMP positive opinion date | 21 July 2022 | Alnylam B V — Amvuttra EMA EPAR public assessment report (EM, p.10 |
Source documents
Explore the full evidence
All 1525 cited findings for Amvuttra — searchable, comparable and answerable in plain language — are in the Neurology research library →
Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.