fazirsiran

Fazirsiran is a potential first-in-class investigational RNA interference (RNAi) therapy, specifically a small interfering RNA (siRNA), designed to reduce the production of mutant alpha-1 antitrypsin protein (Z-AAT). It targets the underlying cause of liver disease in patients with alpha 1-antitrypsin deficiency (AATD) by inhibiting hepatic synthesis of Z-AAT, which accumulates in hepatocytes and leads to progressive liver fibrosis. Fazirsiran has demonstrated significant reductions in both serum and hepatic Z-AAT levels, improvements in histological measures such as globule burden and fibrosis regression, and favorable safety profiles in clinical trials. The drug is being developed for the treatment of AATD-associated liver disease and has received Breakthrough Therapy Designation and Orphan Drug Designation from the US FDA[1][2][3][4][6].

Other names

fazirsiran sodium