Hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with stage 1 or stage 2 polyneuropathy

Active Ingredient: Vutrisiran

Indication for Vutrisiran

Population group: only adults (18 years old or older)
Therapeutic intent: Curative procedure

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

25 mg once every 3 months

For:

Dosage regimens

Subcutaneous, 25 milligrams vutrisiran, once every 90 days.

Detailed description

The recommended dose is 25 mg administered via subcutaneous injection once every 3 months.

Vitamin A supplementation at approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients treated with vutrisiran.

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 is missed, vutrisiran should be administered as soon as possible. Dosing should be resumed every 3 months, from the most recently administered dose.

Elderly patients

No dose adjustment is required in patients ≥65 years of age.

Dosage considerations

The subcutaneous injection should be administered into one of the following sites: the abdomen, thighs, or upper arms. It should not be injected into scar tissue or areas that are reddened, inflamed, or swollen.

If injecting into the abdomen, the area around the navel should be avoided.

Active ingredient

Vutrisiran

Vutrisiran is covalently linked to a ligand containing three N-acetylgalactosamine (GalNAc) residues to enable delivery of the siRNA to hepatocytes. Through a natural process called RNA interference (RNAi), vutrisiran causes the catalytic degradation of TTR mRNA in the liver, resulting in the reduction of variant and wild-type serum TTR protein levels.

Read more about Vutrisiran

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