US approves first RNA interference drug

By Anthony King2018-08-16T10:30:00

A photograph of ONPATTRO™ (patisiran) packaging and product vial

Source: © Business Wire

Alnylam’s Onpattro (patisiran) can treat a rare hereditary disease by silencing a specific gene

The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food and Drug Administration (FDA). Alnylam Pharmaceuticals got the green light for Onpattro (patisiran), to treat hereditary transthyretin amyloidosis – a rare, progressive and often fatal disease.