The FDA is poised to approve the first-ever drug that mutes disease-causing genes

AUGUST 7, 2018

FDA decision on first-of-its-kind drug expected soon

The deadline for the FDA to decide whether to approve a first-of-its kind treatment is looming. The drug, patisiran, aims to treat a rare condition called hATTR that can lead to nerve degeneration and heart damage. If approved, it would be the first drug to rely on a technique known as RNA interference, which lets scientists mute genes that aren't working the right way. It's not clear how much the drug will cost if it gets the green light, but the Institute for Clinical and Economic Review has already released a draft report suggesting that assumed price isn’t “economically attractive.” The agency has to announce its decision by tomorrow, but it could come today.