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Alnylam Pharmaceuticals’ treatment for an increasingly common heart disease succeeded in a closely watched clinical trial, the company said Wednesday, clearing the path to approval and affirming the promise of a drug analysts expect to reap blockbuster sales.

Patients treated with the intravenous drug, called patisiran, did significantly better than those taking a placebo on a test of how far they could walk over the course of six minutes, meeting the study’s primary goal. The 12-month trial enrolled 360 patients with ATTR-CM, a progressive heart disease. The so-called six-minute walk test is a widely accepted metric in cardiovascular medicine, one that correlates with whether a given treatment can prevent hospitalization and death in the long term.


Patisiran met its secondary goal of improving scores on a patient-reported questionnaire on quality of life relative to placebo. Fewer patients in the patisiran group died or had a heart attack or stroke, but the difference wasn’t statistically significant. Safety data were similar between the treatment and placebo groups, Alnylam said, and patisiran’s most common side effects were reactions at the site of infusion, joint stiffness, and muscle spasms. The company plans to present detailed data from the study at a scientific meeting next month and submit for Food and Drug Administration approval later this year.

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