Eplontersen sodium at a glance
Medicine class
Transthyretin-directed antisense oligonucleotide
Licensed use
Stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis
Administration
Injection under the skin using a single-use pre-filled pen
Specialist care
Prescribed and supervised by a clinician experienced in amyloidosis
Key safeguard
Vitamin A falls during treatment, requiring monitoring and prescribed supplementation
What is Eplontersen sodium used for?
The licensed use is:
Hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
How does Eplontersen sodium work?
It binds transthyretin messenger RNA in liver cells and promotes its breakdown. This reduces production of variant and normal transthyretin, leaving less circulating protein available to form amyloid deposits. Vitamin A also falls because transthyretin carries retinol-binding protein.
Eplontersen sodium preparations and strengths
Injection
Route: Parenteral
Strengths: 45 mg
How to take Eplontersen sodium
Use the pre-filled pen under the skin only after appropriate training. Follow the supplied instructions for use.
Allow the refrigerated pen to reach room temperature naturally; do not use another warming method.
Check that the solution is clear and colourless to yellow. Do not use it if cloudy, discoloured or containing particles.
Self-injection sites are the abdomen or upper thigh. A trained carer may also use the back of the upper arm.
Avoid the area around the navel and skin that is bruised, tender, red, hard, scarred or damaged.
Each pen is for single use.
Eplontersen sodium side effects
Common or expected effects
- Reduced vitamin A on blood testing
- Vomiting
- Redness, itching or pain at the injection site
- Protein detected in urine
- Cataracts
Get urgent medical advice
- Severe allergy, including throat or tongue swelling, breathing difficulty or collapse
- New night blindness, persistent dry eyes, eye inflammation or marked visual change
Eplontersen sodium in pregnancy
It should not be used during pregnancy because it lowers vitamin A, which is important for fetal development, and human pregnancy data are unavailable. Pregnancy should be excluded before starting, and effective contraception is needed during treatment. Planned or unplanned pregnancy requires prompt specialist review.
Eplontersen sodium while breastfeeding
It is unknown whether eplontersen enters human milk, and risk to a breastfed child cannot be excluded. The prescriber and patient must decide whether to stop breastfeeding or avoid treatment after considering the benefits of each.
Eplontersen sodium interactions
Formal clinical interaction studies have not been performed. Interactions through CYP enzymes, drug transporters or displacement of highly protein-bound medicines are not expected.
Vitamin A-containing supplements
Use only supplementation advised by the specialist; independently increasing vitamin A may be harmful, particularly during pregnancy.
Common questions about Eplontersen sodium
Answers are fully visible for fast scanning and source review.
Can eplontersen sodium be self-injected?
Yes. A patient or carer may administer it after training, with the first patient- or carer-given injection performed under healthcare-professional guidance.
Why is vitamin A needed?
Lowering transthyretin also lowers circulating vitamin A. Specialist-directed supplementation helps reduce the risk of deficiency-related eye symptoms.
What eye symptoms should be reported?
Promptly report reduced night vision, night blindness, persistent dry eyes, eye inflammation or other significant visual changes.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.