Patisiran

Patisiran is a medication used in the treatment of hereditary transthyretin-mediated amyloidosis (hATTR), a rare genetic disorder characterized by the buildup of abnormal amyloid protein in various tissues and organs, leading to organ dysfunction. It belongs to a class of drugs called small interfering RNA (siRNA) therapeutics, which work by interfering with the production of the abnormal protein.

Indications

Patisiran is indicated for the treatment of polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Dosage

The recommended dosage of patisiran is X mg administered as an intravenous infusion once every three weeks.

Contra-Indications

Patisiran is contraindicated in patients with a history of hypersensitivity to the active substance or any of the excipients.

Special Precautions

Special precautions should be taken in patients with renal or hepatic impairment. Close monitoring of renal and hepatic function is recommended during treatment with patisiran.

Side Effects

Common side effects of patisiran may include infusion-related reactions, peripheral edema, and constipation. Serious side effects may include severe allergic reactions, hepatotoxicity, and thrombocytopenia.

Drug Interactions

Patisiran may interact with drugs that are substrates of P-glycoprotein or cytochrome P450 enzymes. Concurrent use of strong inhibitors or inducers of these enzymes may alter the pharmacokinetics of patisiran.