Alnylam Reports Phase 3 Amvuttra Results for Rare Heart Disease ATTR-CM

Alnylam Pharmaceuticals has unveiled promising findings from an advanced clinical trial of Amvuttra (vutrisiran), an RNA interference therapeutic designed to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This condition is characterized by the accumulation of misfolded transthyretin protein within the heart muscle, leading to irreversible cardiovascular damage over time. ATTR-CM is a rapidly progressing and often under-recognized disease impacting over 300,000 individuals worldwide.

Amvuttra, administered quarterly through subcutaneous injections, leverages the body's natural mechanisms to swiftly reduce transthyretin protein levels. In March, it gained approval from the US Food and Drug Administration (FDA) for the treatment of cardiomyopathy associated with both wild-type and hereditary ATTR-CM. The approval was predicated on results from the phase 3 HELIOS-B trial, which demonstrated that Alnylam's therapeutic lowered the risk of all-cause mortality and recurrent cardiovascular incidents by 28% across the study's participants during a double-blind treatment phase lasting up to 36 months.

At the recent Heart Failure Congress hosted by the European Society of Cardiology, new findings from the HELIOS-B trial were unveiled, highlighting Amvuttra's efficacy in reducing critical cardiovascular events, including hospitalizations due to cardiovascular and heart failure issues. These events frequently precede all-cause mortality and serve as vital indicators of the disease's progression. By the 42nd month mark, Amvuttra was shown to decrease the risk of all-cause mortality by 36% and cardiovascular mortality by 33% when compared to a placebo. Furthermore, the frequency of urgent visits due to heart failure was diminished by 46%.

Pushkal Garg, Alnylam's chief medical officer, expressed confidence in the results, stating: “From the primary analysis of HELIOS-B, we know that Amvuttra profoundly impacts ACM, while preserving patients’ functional capacity and quality of life.” Garg emphasized the significance of these findings, particularly in light of the prevalent use of heart failure medications within the study group, reinforcing Amvuttra's status as a clinically superior, first-line treatment option for those suffering from ATTR-CM.

In addition to ATTR-CM, Amvuttra holds existing approvals for treating polyneuropathy stemming from hereditary transthyretin-mediated amyloidosis. The drug has also garnered recommendations from the European Medicines Agency’s human medicines committee for addressing ATTR-CM, with a decisive verdict from the European Commission anticipated by the third quarter of 2025.

These developments underscore Amvuttra's potential as a groundbreaking therapeutic option, offering renewed hope for those affected by this challenging cardiac condition. As Alnylam continues to advance the treatment landscape for ATTR-CM, the growing body of evidence supporting Amvuttra's effectiveness marks a significant stride in managing this debilitating disease. The company's commitment to enhancing patient outcomes is evident in these recent findings, paving the way for expanded use and recognition of Amvuttra in cardiovascular care globally.