Alnylam Presents New Data Supporting Vutrisiran Benefits for ATTR-CM

Findings from ACC.26 conference highlight potential of RNAi therapy for rare heart condition.

Mar. 31, 2026 at 5:13pm

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An ethereal, ghostly X-ray image of the internal structure of a human heart, with delicate lines and glowing red highlights, conceptually representing the promise of new treatments for a rare cardiac disease.New data on Alnylam's RNAi therapy vutrisiran suggests it may offer benefits for patients with the rare heart condition ATTR-CM.Cambridge Today

Alnylam Pharmaceuticals, the leading RNAi therapeutics company, announced new clinical and real-world data from studies of its investigational therapy vutrisiran for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) at the American College of Cardiology's 26th Annual Scientific Session.

Why it matters

ATTR-CM is a rare and serious condition caused by the buildup of abnormal transthyretin protein in the heart, leading to progressive heart failure. The new data presented by Alnylam provides further evidence that vutrisiran, an RNAi therapeutic, may offer benefits for ATTR-CM patients by reducing transthyretin levels and potentially slowing disease progression.

The details

The data included positive results from a Phase 3 clinical trial evaluating vutrisiran's safety and efficacy, as well as real-world evidence on the therapy's use. Alnylam reported that vutrisiran demonstrated statistically significant improvements in measures of cardiac function, quality of life, and other outcomes for ATTR-CM patients compared to placebo.

  • The data was presented at the American College of Cardiology's 26th Annual Scientific Session (ACC.26) held from March 28-30, 2026.

The players

Alnylam Pharmaceuticals

The leading RNAi therapeutics company that developed the investigational therapy vutrisiran for ATTR-CM.

Vutrisiran

An RNAi therapeutic being evaluated by Alnylam for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

ATTR-CM

A rare and serious condition caused by the buildup of abnormal transthyretin protein in the heart, leading to progressive heart failure.

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What they’re saying

“These new data further support the potential of vutrisiran to provide meaningful benefits for patients living with this devastating disease.”

— Akshay Vaishnaw, President of R&D and Chief Medical Officer at Alnylam

What’s next

Alnylam plans to submit a New Drug Application to the U.S. Food and Drug Administration for vutrisiran later this year.

The takeaway

The positive results from Alnylam's studies of vutrisiran highlight the promise of RNAi therapies in treating rare and serious conditions like ATTR-CM, which currently have limited treatment options available.