STAT

FDA approves first-ever RNAi-based therapy

The FDA approved the first-ever therapy based on a technique known as RNA interference.
Source: Kristoffer Tripplaar/Sipa USA/AP

The Food and Drug Administration on Friday approved a landmark rare disease treatment — the first to rely on a Nobel-prize-winning technique known as RNA interference, which silences disease-causing genes.

The approval is a major accomplishment for Cambridge, Mass.-based Alnylam, which will be marketing the drug, patisiran, as Onpattro and which has been working to bring an

You’re reading a preview, subscribe to read more.

More from STAT

STAT1 min read
STAT+: Health Care Leaders Plot How To Expand Diversity In Clinical Trials
Experts gathered at the Milken Institute Global Conference this week to discussed potential strategies to expand diversity in clinical trials. Here are some of their big ideas.
STAT1 min read
STAT+: Senate Drug Shortage Bill Would Pay Hospitals Bonuses For Good Contracting Practices
A bipartisan Senate bill takes a new approach to persistent drug shortages: have Medicare pay bonuses to hospitals and physicians for contracting that ensures a steady supply.
STAT2 min readCrime & Violence
STAT+: Pharmalittle: We’re Reading About A Medicare Price-negotiation Ruling, Pharma’s Slipping Reputation, And More
A U.S. judge dealt a blow to two drugmakers challenging the authority for Medicare to negotiate the prices of prescription drugs.

Related Books & Audiobooks