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author：knowledge 2025-10-01 22:49:52 - browse：53

Alnylam headquarters — Kristoffer Tripplaar/Sipa USA/AP

The Food and Drug Administration rejected an Alnylam Pharmaceuticals’ therapy for an increasingly prevalent heart disease, the company said Monday, citing insufficient evidence that the treatment meaningfully improved patients’ lives.

Alnylam will no longer seek approval of its drug, an intravenous medicine sold as Onpattro, for people with ATTR-CM, a progressive disease that leads to heart failure if left untreated. The company is in the late stages of development of an updated version of Onpattro that can be administered through a small needle under the skin. Data from a pivotal study, expected next year, would support an FDA application for that treatment.

For Onpattro, a 12-month clinical trial found that the treatment outperformed placebo on a test of how far patients could walk over the course of six minutes, suggesting the medicine slowed down the progression of ATTR-CM. Onpattro also improved patients’ scores on a quality-of-life questionnaire compared to placebo.

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