Eplontersen

Eplontersen

Eplontersen

What is eplonters?

Eplontersen belongs to a class of drugs called LICA (ligand-conjugated antisense oligonucleotide). Eplontersen reduces the amount of TTR protein produced, which slows disease progression and improves neuropathy and the patient's quality of life.

Eplontersen is self-administered monthly as a subcutaneous injection.

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

What is ATTR amyloidosis?

ATTR amyloidosis is caused by a protein in the body called transthyretin (TTR) that changes its normal shape and then forms fibrous clumps. These misshapen clumps of protein then build up in various organs and nerves, which can cause the organs and nerves to not function properly.

ATTR amyloidosis polyneuropathy (ATTR-PN) involves the buildup of amyloid protein in nerves and can cause symptoms such as loss of sensation, tingling, numbness, or pain in the hands and feet.

ATTR cardiomyopathy (ATTR-CM) is when amyloid clumps build up in the heart tissue, impairing the heart's ability to function and causing symptoms similar to heart failure and an enlarged heart.

Eplontersen side effects

Common side effect of eplontersenCauses included low serum vitamin A levels and vomiting, which occurred in at least 9% of patients. Low vitamin A levels are a serious but common side effect of treatment with this medicine. Your doctor should instruct you to take vitamin A supplements while using Eplontersen. Do not take more than the amount of vitamin A recommended by your doctor. Call your doctor if you experience eye problems such as difficulty seeing at night or in poorly lit areas (night blindness) or dry eyes. If you experience eye problems while taking this medicine, your doctor should send you to an ophthalmologist.

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

Before you take this medicine

pregnancy

Tell your doctor if you are pregnant, planning to become pregnant, or think you may be pregnant, as it is not known whether Eplontersen can harm your unborn baby. Changes in vitamin A levels and vitamin A supplementation associated with the use of eplontersen may harm your unborn baby.

Breastfeeding

Tell your doctor if you are breast-feeding or plan to breast-feed. It is not known whether eplontersen can pass into breast milk or harm your baby. Talk to your doctor about the best way to feed your baby while using this medicine.

How should I use Eplontersen?

Read the detailed instructions for use included with your Eplontersen single-dose autoinjector. Your doctor will show you or your caregiver how to inject eplontersen.

If you or your caregiver have any questions, contact your doctor.

Instructions for use

Remove the single-dose autoinjector from the refrigerator 30 minutes before the injection and allow it to warm to room temperature. Do not use other heating methods.

Visually inspect the autoinjector for particles and discoloration before administration. The solution should appear colorless to yellow. Do not use if cloudiness, particulate matter, or discoloration is observed prior to administration.

Eplontersen will be injected by you or a caregiver under the skin (subcutaneously) into your stomach area (abdomen) or into the front of your thighs (thighs). A nurse may also give you an injection into the outer area of ​​your upper arm.

Eplontersen should be injected once on the same day of each month

Follow your doctor's instructions at the time of injecting Eplontersen.

For more detailed instructions with diagrams click here. Wainua Patient Information

If you miss a dose, take the missed dose as soon as possible. Then, inject WAINUA one month after your last dose to return to a monthly dosing schedule. If you have questions about your schedule, contact your doctor.

Dosage information

Recommended Eplontersen Dose: 45 mg administered by subcutaneous injection once a month.

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

How effective is Eplontersen?

In the global, open-label, randomized phase 3 NEURO-TTRansform study of eplontersen in patients with ATTR amyloidosis polyneuropathy (ATTRv-PN), there was an 81.2% reduction in serum transthyretin (TTR) concentration compared to baseline. Eplontersen reduced TTR protein production.

The study showed that eplontersen had a statistically significant effect on the modified neuropathy impairment score +7 (mNIS+7), which is a measure of the progression of a neuropathic disease, compared to the placebo group.

Treatment also significantly improved patient-reported quality of life compared to the external placebo group (p<0.0001), as measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).

Eplontersen is also being studied for the treatment of ATTR cardiomyopathy (ATTR-CM).

Interactions

Tell your doctor about all medications you take, including prescription and over-the-counter medications, vitamins, and herbal supplements.

Tell your doctor if you take:

• Vitamin-A- oder Beta-Carotin-Ergänzungsmittel.

Ask your doctor or pharmacist if you are not sure whether you are taking any of these medicines. Find out about the medications you are taking. Keep a list of these to show your doctor or pharmacist when you get a new medicine. Not all possible interactions are listed here.

Ingredients

Active ingredient:Eplontersen sodium.

Wainua Inactive Ingredients:dibasic sodium phosphate, anhydrous; monobasic sodium phosphate, dihydrate; sodium chloride; Water for injections and may contain hydrochloric acid and sodium hydroxide for pH adjustment.

storage

• Im Originalkarton im Kühlschrank bei 2 °C bis 8 °C lagern.
• Wainua kann im Originalkarton auch bis zu 6 Wochen lang bei einer Raumtemperatur von nicht mehr als 30 °C aufbewahrt werden.
• Lassen Sie Wainua keine Temperaturen über 30 °C erreichen.
• Wenn Sie einen bei Raumtemperatur gelagerten Autoinjektor nicht innerhalb von 6 Wochen verwenden, entsorgen Sie ihn.
• Nicht einfrieren.
• Setzen Sie WAINUA weder Hitze noch Licht aus.

Pursue

Distributed by:AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850 AstraZeneca.

Ionis Pharmaceuticals, Inc.

References

1. Ionis präsentiert positive Ergebnisse der Phase-3-Studie NEURO-TTRansform auf dem International Symposium on Amyloidosis
2. Wainua-Produktetikett der Food and Drug Administration (FDA).

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