The first gene-silencing drug wins FDA approval

A Nobel Prize–winning discovery — that small double-stranded RNA molecules can silence genes by interrupting the translation of DNA’s instructions into proteins — is finally delivering on its medical promise.

The first drug that takes advantage of this natural biological process, called RNA interference, was approved August 10 by the U.S. Food and Drug Administration. It targets a rare hereditary disease that causes misshapen proteins to build up in patients’ nerves, tissues and organs, causing loss of sensation, organ failure and even death.

Hereditary transthyretin amyloidosis, or ATTR, affects about 50,000 people worldwide. This drug will help the subset of those patients who have neurological impairments.