following an abbreviated submission:
eplontersen (Wainzua®) is accepted for use within NHSScotland.
Indication under review: for the treatment of hereditary transthyretin-mediated
amyloidosis (ATTRv amyloidosis) in adult patients with Stage 1 and 2 polyneuropathy.
Eplontersen offers an additional treatment choice of transthyretin (TTR) gene silencer for
this indication.
Another TTR gene silencer was accepted for use under the ultra-orphan process.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme
(PAS) arrangement delivering the cost-effectiveness results upon which the decision was
based, or a PAS/ list price that is equivalent or lower.
Download detailed advice282KB (PDF)
Medicine details
- Medicine name:
- eplontersen (Wainzua)
- SMC ID:
- SMC2755
- Indication:
For treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (ATTRv).
- Pharmaceutical company
- AstraZeneca UK Ltd
- BNF chapter
- Central nervous system
- Submission type
- Abbreviated
- Status
- Accepted
- Date advice published
- 07 April 2025