Antisense oligonucleotide (TTR silencer)

Eplontersen

Brand names: Wainzua, Wainua (US)

Adult dose

Dose: 45mg SC monthly (autoinjector)

Route: Subcutaneous

Frequency: Monthly

Clinical pearls

Hereditary transthyretin-mediated amyloid polyneuropathy (and emerging cardiomyopathy data)
Specialist neurology / cardiology amyloid centres
NICE TA evaluation; ESC ATTR-CM guidance
Vitamin A 3000 IU/day supplementation per SmPC

Contraindications

Vitamin A deficiency (correct first)
Hypersensitivity

Side effects

Vitamin A deficiency (TTR carries retinol — supplement vitamin A)
Injection-site reactions
Glomerulonephritis (class)
Thrombocytopenia (class)

Interactions

Anticoagulants (theoretical — monitor)

Monitoring

Vitamin A
Renal function
Platelets
Polyneuropathy assessment (mNIS+7)

Reference: BNF; NICE TA evaluation; ESC amyloid cardiomyopathy guidance; SmPC; https://bnf.nice.org.uk/drugs/eplontersen/. Verify against your local formulary and the latest BNF before prescribing.

Curated clinical cross-links plus same-class fallbacks.

Pathways