Alnylam B V — Amvuttra EMA EPAR public assessment report (EMEA/H/C/005852, 2022)
Alnylam B V · 2022 · EMA EPAR public assessment report · company profile →
Report summary
The EMA's public assessment report details the positive opinion for Alnylam's Amvuttra (vutrisiran) for adult patients with stage 1 or 2 hATTR amyloidosis polyneuropathy. The report covers the drug's quality, non-clinical profile, and clinical pharmacology, confirming its mechanism as a second-generation siRNA. The pivotal HELIOS-A study demonstrated significant improvements in neurological endpoints (mNIS+7, Norfolk QoL-DN, 10-MWT) and quality of life compared to placebo, and non-inferiority to patisiran in TTR reduction. Safety findings included common adverse events like injection site reactions, arthralgia, and dyspnoea, with no new major safety concerns identified. The overall benefit-risk balance was deemed positive.
Key findings — cited to the page
| Incidence of Infusion Related Reactions (IRRs) | 10 (23.8%) n (%) | HELIOS-A patisiran · p.154 |
| Vutrisiran subjects with Grade 3 chronic kidney disease | 2 subjects | HELIOS-A · p.125 |
| Patisiran subjects with Grade 3 chronic kidney disease | 3 subjects | HELIOS-A · p.125 |
| All vutrisiran group subjects with Grade 3 abnormalities in coagulation parameters | 3 subjects | · p.128 |
| Vutrisiran patients with Grade 3 abnormalities in chemistry parameters | 4 patients | · p.128 |
| Vutrisiran subjects with Grade 3 haematology abnormalities | 2 subjects | · p.127 |
| Infusion related reactions (IRRs) incidence (patisiran) | 23.8 % | HELIOS-A · p.120 |
| Indication | treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy | Amvuttra (vutrisiran) · p.8 |
| Marketing authorisation application submission date | 10 September 2021 | Amvuttra (vutrisiran) · Europe · p.8 |
| Clinical dose | 25 mg | vutrisiran · p.41 |
| CHMP positive opinion date | 21 July 2022 | Amvuttra (vutrisiran) · p.10 |
| Maximum recommended patisiran dose for patients ≥100 kg | 30 mg | HELIOS-A · p.46 |
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 →