Eplontersen sodium: clinical use

Prescribing considerations

• Prescribe and supervise within a service experienced in amyloidosis management.
• Correct low serum vitamin A and assess deficiency symptoms before initiation.
• Exclude pregnancy before initiation and ensure effective contraception where relevant.
• Evidence is unavailable in children, after liver transplantation, severe renal impairment, or moderate-to-severe hepatic impairment; assess benefit and risk carefully.

Contraindications and cautions

• Hypersensitivity to eplontersen or any excipient.
• Avoid use during pregnancy because of the potential risk from disturbed vitamin A levels.

Monitoring

• Serum vitamin A before treatment and when clinically indicated.
• Ocular symptoms suggestive of vitamin A deficiency; arrange ophthalmology assessment when present.
• Pregnancy status and contraception where relevant.
• Clinical review of proteinuria or renal concerns when identified.

Clinical pharmacology

A GalNAc-conjugated antisense oligonucleotide delivered preferentially to hepatocytes. It degrades variant and wild-type transthyretin messenger RNA, reducing hepatic transthyretin synthesis. Nucleases metabolise it, and short oligonucleotide metabolites are cleared renally.

Formulation and product differences

• The product is a ready-to-use, single-use pre-filled pen containing a clear, colourless-to-yellow solution for subcutaneous injection.
• It is essentially sodium-free and must not be mixed with other medicinal products.

Injection

Route: Parenteral

Strengths: 45 mg