Hereditary transthyretin-mediated amyloidosis with stage 1 or stage 2 polyneuropathy

Active Ingredient: Eplontersen

Indication for Eplontersen

Population group: only adults (18 years old or older)

Therapeutic intent: Curative procedure

Eplontersen is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.

For this indication, competent medicine agencies globally authorize below treatments:

45 mg once monthly

For:

Dosage regimens

Subcutaneous, 45 milligrams eplontersen, once monthly.

Detailed description

The recommended dose of eplontersen is 45 mg administered monthly.

Vitamin A supplementation at approximately, but not exceeding, 2 500 IU to 3 000 IU vitamin A per day is advised for patients treated with eplontersen.

Treatment should be initiated as early as possible after symptom onset.

The decision to continue treatment in those patients whose disease progresses to stage 3 polyneuropathy should be taken at the discretion of the physician based on the overall benefit and risk assessment.

Missed dose

If a dose of eplontersen is missed, then the next dose should be administered as soon as possible. Dosing should be resumed at monthly intervals from the date of the last dose; a double dose should not be administered.

Dosage considerations

If self-administered, eplontersen should be injected in the abdomen or upper thigh region. If a caregiver administers the injection, the back of the upper arm can also be used.

Eplontersen should not be injected into skin that is bruised, tender, red, or hard, into scars or damaged skin, the area around the navel should be avoided.

Active ingredient

Eplontersen
Eplontersen is a N-acetylgalactosamine (GalNAc)-conjugated 2′-O-2-methoxyethyl-modified chimeric gapmer antisense oligonucleotide (ASO) with a mixed backbone of phosphorothioate and phosphate diester internucleotide linkages. The GalNAc conjugate enables targeted delivery of the ASO to hepatocytes. The selective binding of eplontersen to the transthyretin (TTR) messenger RNA (mRNA) within the hepatocytes causes the degradation of both mutant and wild type (normal) TTR mRNA. This prevents the synthesis of TTR protein in the liver, resulting in significant reductions in the levels of mutated and wild type TTR protein secreted by the liver into the circulation. Read more about Eplontersen

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