EC Approves Vutrisiran for ATTR-CM Treatment

The European Commission has granted approval for Alnylam Pharmaceuticals’ therapy, Amvuttra (vutrisiran), aimed at treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults. This marks a significant milestone as Amvuttra becomes the first and only RNA interference (RNAi) therapeutic in the European Union approved for both cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis, specifically hereditary transthyretin-mediated amyloidosis (hATTR).

The approval is based on the robust results from the HELIOS-B Phase III clinical trial. This trial successfully reached all ten pre-specified primary and secondary objectives, highlighting Amvuttra's efficacy. The therapy demonstrated a remarkable 28% reduction in the primary composite outcome of all-cause mortality and recurrent cardiovascular events compared to a placebo. Additionally, there was a notable 36% decrease in mortality observed over a 42-month period, as noted in a secondary endpoint analysis. These outcomes were coupled with improvements in patients’ health status, heart failure symptoms, and functional capacity.

ATTR-CM is a condition resulting from the accumulation of misfolded transthyretin (TTR) fibrils, which can lead to significant cardiovascular damage and increased risk of premature death. For patients within the European Union, Amvuttra is administered via subcutaneous injection every three months. This mode of administration is part of the innovative approach of the therapy, which offers a patient-friendly dosing schedule that does not compromise efficacy.

In addition to its approval in Europe, Amvuttra has already received the go-ahead from the US Food and Drug Administration and the Brazilian Health Regulatory Agency in March 2025. Alnylam Pharmaceuticals is actively working on extending its global reach by submitting applications in various countries to make this therapy widely accessible.

Pushkal Garg, Alnylam Pharmaceuticals’ Chief Medical Officer, emphasized the established efficacy and safety profile of Amvuttra, which is supported by over 6,000 patient-years of global experience in treating hATTR with polyneuropathy. Garg highlighted the therapy's ability to rapidly and sustainably reduce TTR levels through quarterly dosing, offering a clinically distinct approach with the potential to drastically improve outcomes for patients suffering from this severe and potentially life-threatening disease.

In 2024, Alnylam reported positive topline results from a late-stage study of vutrisiran for treating ATTR-CM, reinforcing its therapeutic promise. These findings further underscore Amvuttra’s role in the innovative landscape of treatments targeting ATTR-CM.

The advancement of Amvuttra acts as a catalyst for further developments in the treatment options for ATTR amyloidosis, offering new hope to patients affected by this challenging condition. As the company continues to pursue global submissions, the impact of this RNAi therapeutic is anticipated to expand, providing a critical tool in the management of cardiomyopathy and polyneuropathy associated with ATTR amyloidosis.