A medication for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis.
An RNA interference therapeutic agent that inhibits the production of hepatic transthyretin.
It has a lipid nanoparticle delivery system and targets three untranslated regions of TTR messenger RNA in the liver to reduce circulating, transthyretin protein levels in both variant and wild type ATTR.
Trade name Onpattro.
Hereditary transthyretin-mediated amyloidosis is a fatal rare disease that is estimated to affect 50,000 people worldwide.
It is a small interfering RNA-based drug that is gene silencing, interfering with the production of an abnormal form of transthyretin.
Requires long term administration, which is associated with continued exposure to pre-medication with the glucocorticoids and anti-histamines.
In a trial period of 12 months, Patisiran resulted in preserved functional capacity in patients with transthyretin cardiac amyloidosis (Maurer MS).
As with all agents that suppress hepatic TTR protein synthesis, vitamin A supplementation is recommended to prevent vitamin A deficiency due to impaired vitamin A transport that occurs with reducing circulating TTR levels.