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FDA panel backs Alnylam drug despite doubts over benefit

September 13, 2023


Advisers to the Food and Drug Administration on Wednesday endorsed Alnylam Pharmaceuticals’ drug Onpattro for a rare heart condition, despite questions from agency scientists over the degree of benefit offered by the RNA-based therapy.

The advisory committee’s 9-3 vote sets up a decision by the regulator on the drug’s approval for the condition, which is caused by buildup of a misfolded protein that damages the heart and other organs. In 2018, the FDA approved Onpattro to treat nervous system damage caused by the same toxic buildup of the protein, called transthyretin or TTR.

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