The European Commission (EC) has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) to treat wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults.
Amvuttra is the first and only RNA interference (RNAi) therapeutic approved by the EC for both the cardiomyopathy and the polyneuropathy manifestations of ATTR amyloidosis and hereditary transthyretin-mediated amyloidosis (hATTR).
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The approval is grounded in the positive HELIOS-B Phase III trial outcomes, which demonstrated that the therapy met all ten pre-specified primary and secondary goals.
This includes minimisations in all-cause death and recurrent cardiovascular events, along with improvements in health status, heart failure symptoms and functional capacity.
In the overall population, the therapy has achieved a 28% decrease in the primary composite of all-cause death and recurrent cardiovascular events against placebo.
A significant 36% decrease in death was observed through 42 months in a pre-specified secondary endpoint analysis.
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By GlobalDataATTR-CM occurs due to the accumulation of misfolded transthyretin (TTR) fibrils, leading to severe cardiovascular damage and premature mortality.
In the European Union, the therapy is administered subcutaneously once every three months.
The European Medicines Agency’s Committee for Orphan Medicinal Products endorsed the maintenance of Amvuttra’s orphan designation for ATTR amyloidosis in May 2025.
Following approvals by the US Food and Drug Administration and the Brazilian Health Regulatory Agency in March 2025, the company is progressing with global submissions to make the therapy accessible globally.
Alnylam Pharmaceuticals chief medical officer Pushkal Garg stated: “Amvuttra is supported by a well-established efficacy and safety profile, with over 6,000 patient-years of global experience in the treatment of hATTR with polyneuropathy.
“By delivering rapid and sustained knockdown of TTR through convenient, quarterly dosing, it offers a clinically differentiated approach with the potential to transform outcomes for patients living with this debilitating and potentially fatal disease.”
In 2024, the company shared positive topline outcomes for the late-stage study of vutrisiran for the treatment of ATTR-CM.
