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Antisense oligonucleotide (TTR silencer)

Eplontersen

Brand names: Wainzua, Wainua (US)

Eplontersen is an antisense oligonucleotide used to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Dosing — being independently re-sourced

ClinCalc Pro is rebuilding its dose data from primary open sources — the manufacturer SmPC (eMC), the WHO Model Formulary and other official references — under clinician review. This drug's structured dose is not yet published here. Confirm all doses against the product SmPC and your local formulary before prescribing.

Clinical monograph

How it works

It is a ligand-conjugated antisense oligonucleotide that binds transthyretin messenger RNA in the liver, reducing production of both mutant and wild-type transthyretin protein.

Prescribing in practice

  • Reducing transthyretin lowers serum vitamin A, so vitamin A supplementation is recommended during treatment.
  • It is given by subcutaneous injection at regular intervals, including via a self-administration option.
  • Monitor for injection-site reactions and assess ongoing neurological status.

Monitoring

Monitor neurological function and nutritional status, with vitamin A supplementation and ophthalmic review if symptoms of deficiency arise.

Counselling the patient

  • Take the recommended vitamin A supplement as directed.
  • Report any visual problems, particularly difficulty seeing in dim light.
  • Injection-site reactions can occur; rotate sites and report any that are severe or persistent.

Evidence & guidelines

Efficacy in hereditary transthyretin-mediated polyneuropathy was demonstrated in the NEURO-TTRansform study showing reduced transthyretin and neurological benefit.

Reference: NICE TA evaluation; ESC amyloid cardiomyopathy guidance; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

Pathways