Alnylam gets first RNAi drug approved

Twenty years after the scientific co-founders of Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) discovered RNAi, a drug using the modality has been approved. On Friday, FDA approved Onpattro patisiran to treat polyneuropathy caused by hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) in adults. The indication is narrower than what Alnylam originally sought -- general treatment of hATTR -- in its NDA for Onpattro, and does not include cardiac data.

Alnylam said it plans to launch Onpattro within 48 hours at an average annual list price of $450,000. The company said the effective net price after mandatory government discounts would be $345,000, which translates into a 23.1% discount -- the minimum discount required under the Medicaid best price rule...