Products
Patisiran is marketed as a concentrate for the preparation of an [infusion solution>infusion] (Onpattro). It was approved in the US and EU in 2018 and in many countries in 2019.
Structure and properties
Patisiran belongs to the small interfering RNA (siRNA) group of drugs. It is a small, double-stranded ribonucleic acid. The active ingredient is present in the drug as patisiran sodium. Patisiran is contained in a lipid nanoparticle to ensure uptake (delivery) into liver cells.
Effects
Patisiran (ATC N07XX12) reduces the natural and the mutant TTR protein (transthyretin) in liver cells. Mutations in this gene are the cause of misfolding of the protein and trigger hereditary transthyretin amyloidosis. The effects are due to the so-called RNA interference (RNAi). Patisiran leads to catalytic degradation of TTR mRNA, which inhibits protein synthesis. It binds to a conserved region in the untranslated region of mRNA. The mean half-life is approximately 3.2 days.
Indications
For the treatment of hereditary transthyretin amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or 2 polyneuropathy.
Dosage
According to the SmPC. The drug is administered as an intravenous infusion every three weeks.
Contraindications
- Severe hypersensitivity
Full precautions can be found in the drug label.
Adverse effects
The most common potential adverse effects include peripheral edema and infusion-related reactions.
