In first, FDA approves RNA interference drug from Alnylam
The FDA granted the first U.S. approval of a drug that works by “silencing” genes to stop disease. Alnylam Pharmaceuticals’ drug, Onpattro, stems from Nobel Prize-winning research concerning a cellular process known as RNA interference, which can be used to interrupt the production of disease-causing proteins by degrading the signals that convey genetic instructions.
Onpattro, which was approved to treat peripheral nerve disease caused by hereditary ATTR amyloidosis, won’t come cheap as the company plans to sell it at an average annual list price of $450,000 per patient.
