RNAi (siRNA) therapeutics
Neurology· 2 reviewed drugs· 2319 cited findings behind them
What the regulatory reviews cover
This library includes two RNAi (siRNA) therapeutics that have undergone regulatory review: Amvuttra and Onpattro. Amvuttra was reviewed for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy, and for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Onpattro was reviewed for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
The regulatory documents report various outcomes from clinical trials. For Amvuttra, the NIS LS mean difference (vutrisiran – placebo) was -22.75. For Onpattro, the mNIS+7 LS Mean Difference (Patisiran-LNP - Placebo) was -30.63. It is important to note that these drugs were studied in different trials, and therefore, the reported figures are not directly comparable head-to-head.
The drugs — one cited result each
| Amvuttra Alnylam Pharmaceuticals, Inc. | NIS LS mean difference (vutrisiran – placebo): -22.75 Alnylam B V — Amvuttra EMA EPAR public assessment report (EM, p.77 |
| Onpattro Alnylam Pharmaceuticals, Inc. | mNIS+7 LS Mean Difference (Patisiran-LNP - Placebo): -30.63 Alnylam B V — Onpattro EMA EPAR public assessment report (EM, p.98 |
Different drugs were studied in different trials — these figures are not head-to-head comparisons.
Explore the full evidence
Every finding behind these drugs — searchable, comparable and answerable in plain language — is in the Neurology research library →
Lists reflect the regulatory review documents in this library, not all therapies that exist. Educational reference only — not medical or investment advice.