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 RNAi-based therapy to market for more than a decade.
Unlock this article by subscribing to STAT Plus. To get you started, enjoy 50% off your first 3 months!
GET STARTEDWhat is it?
STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.
What's included?
- Daily reporting and analysis
- The most comprehensive industry coverage from a powerhouse team of reporters
- Subscriber-only newsletters
- Daily newsletters to brief you on the most important industry news of the day
- Online intelligence briefings
- Frequent opportunities to engage with veteran beat reporters and industry experts
- Exclusive industry events
- Premium access to subscriber-only networking events around the country
- The best reporters in the industry
- The most trusted and well-connected newsroom in the health care industry
- And much more
- Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
About the Author
There are volumes of cases of Friedreich’s ataxia in Louisiana. How do we get the word out to neurologist here?