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author：focus 2025-08-06 04:47:18 - browse：32

Alnylam headquarters — Kristoffer Tripplaar/Sipa USA/AP

A group of advisers to the Food and Drug Administration voted in favor of Alnylam Pharmaceuticals’ treatment for a debilitating heart disease Wednesday, but only after a day-long debate challenging whether the drug’s modest observed effects were actually meaningful for patients.

The group of independent experts voted 9-3 that the benefits of Alnylam’s drug, called patisiran, outweighed its risks for patients with ATTR-CM, a progressive disease that leads to fatal heart failure if left untreated. The FDA, which is not required to follow the advice of advisers, is expected to make a final decision on patisiran by Oct. 8.

“It’s like a light wind blowing in favor of patisiran over placebo,” said David Cella, a neurologist at the Northwestern University Feinberg School of Medicine who voted in favor of patisiran. David Moliterno, a cardiologist at the University of Kentucky, added that “the good news in this story is that while there may be a small benefit, it doesn’t look like there’s a signal for harm.”

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