First RNA interference drug on the steps of FDA approval

Positive results in a clinical trial using patisiran, a RNA interference (RNAi) drug, illustrates that the B-SMART consortium is on the right track: Similar to the therapeutics addressed in B-SMART, patisiran is a therapeutic that silences a disease-causing protein  in patients with hereditary amyloidosis (hATTR), and showed excellent result in a phase 3 clinical study. The developer, the US-American pharmaceutical company Alnylam, hopes to file for approval in the U.S. by the end of the year, which would make patisiran the first RNAi therapeutic on the market. Administration in the Alnylam trial was by intravenous injection. The particles used are virtually the same lipid nanocarriers as are used in B-SMART, and have been shown to also work by local brain injection. If approved, sales of patisiran are expected to reach $1 billion sales in 2023.

More information on the US study can be found here.