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This website is intended for UK Healthcare Professionals only. Other UK residents please visit astrazeneca.co.uk
Once-monthly WAINZUA significantly lowers serum TTR levels, reduces neuropathy impairment and improves quality of life vs. external placebo in adult patients with Stage 1 or 2 ATTRv amyloidosis with polyneuropathy.1,2
*WAINZUA can be directly administered by patients, caregivers or HCPs at home with a monthly delivery. The first injection adminsitered by the patient or caregiver should be performed under the guidance of an appropriately qualified healthcare professional. Patients and/or caregivers should be trained in the subcutaneous administration of WAINZUA.1
In November 2024, NICE published its Technology Appraisal Guidance recommendations for eplontersen.3
RECOMMENDATIONS:
1.1 Eplontersen is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy. It is only recommended if the company provides eplontersen according to the commercial arrangement.3
1.2 Use the least expensive option of the available
treatments (including eplontersen and vutrisiran).
Take account of administration costs, dosages, price
per dose and commercial arrangements. If the least
expensive option is unsuitable, people with the condition and their healthcare professional should discuss the advantages and disadvantages of other treatments.3
Alongside its clinical trial evidence, a cost comparison suggests that eplontersen is cost saving compared with vutrisiran.*3
*There are no clinical trials directly comparing WAINZUA with vutrisiran. An indirect comparison suggests that WAINZUA works as well as vutrisiran.3
In April 2025, the Scottish Medicines Consortium (SMC) published its detailed advice document (DAD) for eplontersen.4
SUMMARY:
WAINZUA (eplontersen) is accepted for use within NHSScotland for the treatment of ATTRv amyloidosis in adult patients with Stage 1 and 2 polyneuropathy.4
Eplontersen offers an additional treatment choice of TTR gene silencer for this indication.4
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.4
ATTR=transthyretin amyloidosis; ATTRv=hereditary transthyretin amyloidosis; ATTRv-PN=hereditary transthyretin amyloidosis with polyneuropathy; DAD=detailed advice document; NICE=National Institute for Health and Care Excellence; SMC=Scottish Medicines Consortium; TTR=transthyretin.
GB-62842 | November 2025
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This website was created for UK HCPs and has been designed to provide information to educate, empower and enable them in the great work they are doing for patients across a range of therapy areas. This website is intended for doctors, nurses, and pharmacists in the UK. Other UK residents please visit astrazeneca.co.uk.
