Transthyretin amyloidosis (ATTR) is a protein conformational disorder caused by the build-up of transthyretin, the protein carrying thyroxine and retinol in the bloodstream. It can cause ATTR-cardiomyopathy (ATTR-CM) or ATTR-polyneuropathy (ATTR-PN). As effective treatments for ATTR-CM are still limited, Alnylam Pharmaceuticals’ drug vutrisiran is expected to change ATTR-CM treatment paradigm upon market launch in Europe, according to GlobalData, a leading data and analytics company.

Small interfering RNA (siRNA) vutrisiran is currently one of the preferred treatments for ATTR-PN. Cardiologists have been waiting for its approval for the past couple of years, and on 11 June 2025, the drug was approved by the European Commission for ATTR-CM patients.

Costanza Alciati, Pharma Analyst at GlobalData, comments: “Vutrisiran offers a great advantage for ATTR-CM patients. It will be the first transthyretin silencer on the market, as currently available therapies can stabilize the protein, but do not reduce its production.”

The drug will be marketed under the name Amvuttra, as a subcutaneous administration to be given every three months.

Alciati continues: “Vutrisiran is also an easy drug to administered. According to key opinion leaders (KOLs) interviewed by GlobalData, vutrisiran is used in the majority of their ATTR-PN patients.”

Alciati concludes: “It is likely that vutrisiran will become one of the preferred therapies in ATTR-CM as well, at least until the approval of nucresiran, which is currently in clinical trials and is expected to be an improved version of the newly-approved drug. The ATTR-CM space will go through major changes in the next few years, considering the innovative drugs in late-stage pipeline, and hopefully, some of the unmet needs in the space will be mitigated.”