Patisiran and Inotersen in hATTR Amyloidosis

Patisiran (Onpattro) and inotersen (Tegsedi) are TTR mRNA silencing therapies in hereditary ATTR amyloidosis (hATTR). Patisiran is lipid nanoparticle RNAi (siRNA), inotersen is antisense oligonucleotide (ASO). APOLLO and NEURO-TTR trials halted polyneuropathy progression, with regression in some patients. Patisiran 0.3 mg/kg IV every 3 weeks; inotersen 284 mg SC weekly. Patisiran side effects include infusion reactions; inotersen causes thrombocytopenia and glomerulonephritis (requires close monitoring). Vutrisiran (Amvuttra) is next-generation siRNA at 25 mg SC every 3 months. FDA-approved via HELIOS-A. Cardiac hATTR efficacy under investigation.