Eplontersen

Generic name: Eplontersen
Brand names: Wainua
Dosage form: subcutaneous injection (45 mg/0.8 mL)

Usage of Eplontersen

Eplontersen (Wainua) is used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN), which is a rare, progressive disease that can be fatal if not treated. Amyloidosis is a disease caused by a buildup of abnormal proteins called amyloid. 

Eplontersen is from a class of medications called LICA (ligand-conjugated antisense oligonucleotide). Eplontersen works by decreasing the amount of TTR protein that is made, which slows disease progression and improves neuropathy and the patient's quality of life.

Eplontersen is given as a monthly self-administered suBCutaneous injection.

Eplontersen received FDA approval on December 21, 2023, after positive results from the phase III clinical trial NEURO-TTRansform (NCT04136184).

Eplontersen side effects

Common eplontersen side effects were low vitamin A serum levels and vomiting, which occurred in at least 9% of patients. Low vitamin A level is a serious, but common side effect of treatment with this medicine. Your healthcare provider should tell you to take vitamin A supplements while using eplontersen. Do not take more than the amount of vitamin A your healthcare provider has recommended. Call your healthcare provider if you develop eye problems such as difficulty seeing at night, or in low-lit areas (night blindness), or dry eyes. If you develop eye problems while receiving treatment with this medicine, your healthcare provider should send you to see an eye doctor.

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Before taking Eplontersen

Pregnancy

Tell your healthcare provider if you are pregnant, plan to become pregnant or think you may be pregnant, as it is not known if eplontersen can harm your unborn baby. Changes in vitamin A levels and vitamin A supplementation related to use of eplontersen may harm your unborn baby. 

Breastfeeding

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if eplontersen can pass into your breast milk or harm your baby. Talk with your healthcare provider about the best way to feed your baby while you are using this medicine. 

How to use Eplontersen

Recommended eplontersen dose: 45 mg administered by subcutaneous injection once a month.

Eplontersen is available as a 45 mg/0.8 mL single-dose autoinjector.

What other drugs will affect Eplontersen

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. 

Tell your healthcare provider if you take: 

  • Vitamin A or beta-carotene supplements.
  • Ask your healthcare provider or pharmacist if you are not sure if you take any of these medicines. Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine. Not all possible interactions are listed here.

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