Alnylam gets surprise FDA rejection for rare disease drug

In a surprise move, the U.S. Food and Drug Administration rejected a drug made by Alnylam Pharmaceuticals Inc., citing a lack of "clinical meaningfulness."

The agency rejected the advice of its own advisory committee in doing so. Last month, a group of experts voted 9-3 to endorse the drug, patisiran, as a treatment for cardiomyopathy of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare disease that causes a buildup of amyloid proteins outside the cells. Still, clinical meaningfulness of benefit on various metrics was "the subject of rich debate" during the meeting, as Leerink Partners analyst Mani Foroohar wrote in a research note.

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