FDA Approves First RNAi Therapy
The Food and Drug Administration has approved patisiran, the first-ever therapeutic based on RNA interference (RNAi). It treats a rare, progressive polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis, which afflicts about 50,000 people worldwide. Made by the Cambridge, Massachusetts-based Alnylam Pharmaceuticals (press release), patisiran delivers small interfering RNAs (siRNAs) to the liver, where they bind and degrade transthyretin messenger RNA. Transthyretin normally carries vitamin A through the blood, but when mutated, it forms amyloid fibers in cells, causing heart, nervous system, and gastrointestinal tract symptoms that are often fatal. Granted Orphan Drug, Fast Track, Priority Review, and Breakthrough Therapy designations by the FDA to expedite the regulatory process, patisiran is the first drug approved to treat hATTR amyloidosis.
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