On August 10, 2018 the FDA approved patisiran (Onpattro) by Alnylam pharmaceuticals. This is a “first-in-class” drug is a small interfering RNA (siRNA) drug that work inside the cell at the RNA level. The FDA anticipates that [RNA-based Medicine] is the beginning of a new and exciting generation of therapeutics” [1, 2, 5]
The phase 3 trial included 225 patients with a “rare and frequently fatal” hereditary disease called transthyretin-mediated amyloidosis (hATTR), 148 were given the treatment [3]
Onpattro drug formulation contains “lipid excipients (DLin-MC3-DMA, DSPC, cholesterol, and PEG2000-C-DMG)”, because of this registration, the lipid nanoparticle ingredient DSPC (1,2-Distearoyl-sn-glycero-3-phosphocholine), which is also used in Pfizer’s mRNA COVID-19 vaccine as a structural lipid, is thus not conidered as “novel”, unlike the functional lipids ALC-0315 and ALC-0159. [4]