Vutrisiran Sodium

Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) has been approved by the European Commission (EC) to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a potentially fatal disease of the heart muscle. The RNAi therapeutic has been authorised to treat both wild type and hereditary ATTR-CM, which occurs when misfolded transthyretin (TTR) protein accumulates and causes irreversible cardiovascular damage over time. Hereditary ATTR affects approximately 50,000 people worldwide, while wild-type ATTR impacts up to 300,000. Patients may present with cardiomyopathy, polyneuropathy or both manifestations of the disease. Given as a subcutaneous injection once every three months, Amvuttra is designed to work with the body’s natural system to knock down TTR at its source, and is already approved in the EU to treat hereditary TTR-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Alnylam’s chief medical officer, Pushkal Garg, said: “By delivering rapid and sustained knockdown of TTR through convenient, quarterly dosing, [Amvuttra] offers a clinically differentiated approach with the potential to transform outcomes for patients living with this debilitating and potentially fatal disease. “We now look forward to securing access to Amvuttra for eligible patients across the EU as quickly as possible.” The EC’s latest decision on the drug follows a recent recommendation from the European Medicines Agency’s human medicines committee and was based on results from the late-stage HELIOS-B trial. In the overall study population, which included patients receiving Amvuttra as a monotherapy and those using the therapy alongside standard-of-care treatments such as tafamidis and SGLT2 inhibitors, Alnylam’s drug reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% compared to placebo. Mortaliy in this population was also reduced by 36% at month 42 in a pre-specified secondary endpoint analysis, which included up to 36 months of the double-blind period plus six months of open-label extension. “The trial enrolled a broad population reflective of real-world clinical practice, and that’s what makes the results so meaningful,” said HELIOS-B investigator, Marianna Fontana. “This is a milestone for patients, who now have a new treatment option that has the potential to significantly improve outcomes of this disease.”

The therapy achieved a 28% decrease in the primary composite of all-cause death and recurrent cardiovascular events against placebo. Credit: Jo Panuwat D/Shutterstock. 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). 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. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence ATTR-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. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now