ATTR amyloidosis
Neurology· 5 reviewed drugs· 4961 cited findings behind them
What the regulatory reviews cover
This page provides an overview of drugs whose regulatory review documents for ATTR amyloidosis are available in this library. These include Amvuttra, Wainzua, TEGSEDI, Vyndaqel, and Onpattro. Each drug has specific indications, such as the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with polyneuropathy, or wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Different drugs were studied in different trials, and therefore the figures presented are not head-to-head comparisons. For instance, Amvuttra showed an NIS LS mean difference (vutrisiran – placebo) of -22.75. Wainzua demonstrated a maximum TTR expression reduction of 85. TEGSEDI reported a Treatment Difference (LS Mean) in mNIS+7 of -19.7. Onpattro showed a mNIS+7 LS Mean Difference (Patisiran-LNP - Placebo) of -30.63.
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 |
| Wainzua AstraZeneca PLC | Maximum TTR expression reduction: 85 AstraZeneca PLC — Wainzua EMA EPAR public assessment report, p.27 |
| TEGSEDI Ionis Pharmaceuticals, Inc. | Treatment Difference (LS Mean) in mNIS+7: -19.7 AKCEA THERAPS — Tegsedi FDA Review (NDA/BLA 211172, 2026), p.29 |
| Vyndaqel Pfizer Inc. | NIS-LL responders (ITT population): 45.3 Pfizer Inc. — Vyndaqel EMA EPAR public assessment report (EM, p.44 |
| 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.