AstraZeneca PLC — Wainzua EMA EPAR public assessment report (EMEA/H/C/006295, 2025)
AstraZeneca PLC · 2025 · EMA EPAR public assessment report · company profile →
Report summary
The EMA's CHMP has issued a positive opinion for AstraZeneca's Wainzua (eplontersen) for treating hereditary transthyretin-mediated amyloidosis (ATTRv) with polyneuropathy (stages 1 and 2). Eplontersen, an RNA interference therapeutic, demonstrated superior efficacy over placebo in reducing serum TTR concentrations and improving neurological and quality of life scores (mNIS+7 and Norfolk QoL-DN) in the NEURO-TTRansform study. Administered subcutaneously monthly, it achieved over 80% TTR reduction. While generally safe, common adverse events included ocular issues linked to vitamin A deficiency, which are manageable. Eplontersen showed a favorable safety profile compared to inotersen, with lower rates of thrombocytopenia and injection site reactions. The CHMP concluded it is a new active substance and not similar to other orphan ATTRv treatments.
Key findings — cited to the page
| Platelet abnormalities Grade 1a (≥ 100 × 10^9/L to < 140 × 10^9/L) (eplontersen) | 27.8 % | eplontersen · p.123 |
| Shift to Grade 3 in neutrophil counts | 9 % | · p.113 |
| Shift to Grade 3 in lymphocytes counts | 7.6 % | · p.113 |
| Incidence of clinically meaningful platelet count decreases (≥ Grade 1b) | low and similar | eplontersen, external placebo · p.147 |
| Patients with Grade 2 or 3 lymphocyte nadir counts attributed to COVID-19 | 22% | · p.131 |
| Completed Phase 1 ascending dose studies | 2 | eplontersen · p.14 |
| Completed Phase 1 bioequivalence study | 1 | eplontersen · p.14 |
| Phase 3 studies in ATTR-CM | 2 | eplontersen · p.14 |
| Administration frequency | monthly | eplontersen · p.14 |
| Pivotal study name | ION-682884-CS3 (NEURO-TTRansform) | eplontersen · p.14 |
| Long-term extension study name | ION-682884-CS13 | eplontersen · p.14 |
| Legal basis for application | Article 8.3 of Directive 2001/83/EC - complete and independent application | · p.14 |
Source: AstraZeneca PLC ↗ · 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 →