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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