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After a decades-long wait, the FDA is on the brink of approving a landmark rare disease treatment — the first to rely on a Nobel prize-winning technique known as RNA interference, or RNAi.

The widely anticipated approval will be a watershed moment not only for its manufacturer, Alnylam, but for the broader field of research into RNAi, which lets scientists mute genes that aren’t functioning properly. The FDA must announce its decision by Friday to meet a regulatory deadline.


Already, regulators in the European Union and the U.K. have given the product, patisiran, an initial greenlight. Full approval could come by the end of September.

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  • Gee everyone will want this. The current corruption at our regulatory agencies, along with profit incentives related to “Rare Diseases” makes it the perfect time to market this product. Most of the research is Proprietary (secret) anyway. We won’t know of adverse events until long after this stuff has reached market saturation. After all those clever industry insiders believe that Healthcare is a choice, anyone desperate enough to choose to try this will only have themselves to blame.

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