Alnylam B V — Amvuttra EMA EPAR variation assessment report (EMEA/H/C/005852, 2022)
Alnylam B V · 2022 · EMA EPAR variation assessment report · company profile →
Report summary
This report summarizes Alnylam's Type II variation application for Amvuttra (vutrisiran) to extend its indication to include wild-type or hereditary transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM), based on the HELIOS-B Phase 3 study. Vutrisiran's pharmacokinetics and pharmacodynamics were largely unaffected by intrinsic or extrinsic factors. The HELIOS-B trial, involving 655 patients, demonstrated significant reductions in all-cause mortality and recurrent cardiovascular events, improved 6-minute walk test distance, and better quality of life (KCCQ-OS, NYHA class) compared to placebo. Positive effects on cardiac biomarkers were also observed. The safety profile was consistent with previous findings, showing a favorable benefit-risk balance with fewer adverse events in the vutrisiran group.
Key findings — cited to the page
| ATTR-CM etiology in heart failure with preserved ejection fraction | 13 % | · p.9 |
| Prefilled syringes of AMVUTTRA sold worldwide | 13387 syringes | AMVUTTRA · Global · p.105 |
| Study name | HELIOS-B | Vutrisiran · p.6 |
| Patients from Spain | 64 patients | Spain · p.41 |
| Application type | Type II variation | Amvuttra · p.6 |
| New therapeutic indication | Extension of indication to include treatment of wild-type or hereditary transthyretin-mediated amyloidosis in adult patients with cardiomyopathy (ATTR-CM) | Amvuttra · p.6 |
| Patients from United Kingdom | 151 patients | United Kingdom · p.41 |
| HELIOS-B placebo arm patients | 328 patients | · p.14 |
| Patients from Argentina | 16 patients | Argentina · p.41 |
| Orphan medicinal product designation date | 25 May 2018 | Amvuttra · p.6 |
| Withdrawal of market protection request date | 14 February 2025 | AMVUTTRA · p.7 |
| Prevalence of ATTRv-CM | over 40,000 persons | Global · p.9 |
Source: Alnylam B V ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →