Vutrisiran Sodium

MONDAY, March 24, 2025 -- The U.S. Food and Drug Administration (FDA) has approved a new drug for a serious heart condition that affects thousands of people. The drug, called Amvuttra (vutrisiran), is made by Alnylam Pharmaceuticals and is used to treat transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a disease in which harmful proteins build up in the heart, making it harder to pump blood, according to the American Heart Association (AHA). In one study, Amvuttra helped lower the risk of death and heart problems by 28% over three years compared to a placebo. The drug is given as a shot every three months. “We hope this could become our flagship franchise. We’re going to help thousands more patients,” Alnylam CEO Yvonne Greenstreet said in an interview with STAT News . “It’s also a transformational moment for the company.” But the treatment comes at a steep price. Each dose costs about $119,000, or $476,000 per year. That’s more than double the cost of a similar drug from BridgeBio, and more expensive than Pfizer’s pills for the same condition. “There are many, many, many undiagnosed patients and substantial unmet need in this space. This market is very large, underdiagnosed, undertreated,” said Dr. Mani Foroohar , an analyst with the healthcare investment company Leerink. Still, the high cost may make it harder for patients -- especially folks on Medicare Advantage plans -- to get the drug. Private insurers may prefer cheaper options like pills from Pfizer or BridgeBio. Amvuttra works differently from those drugs. It uses RNA technology to stop the body from making the bad protein, while the other treatments work to stabilize the protein before it causes harm. In trials, BridgeBio’s treatment showed a 42% drop in deaths and heart hospital visits over 30 months. Amvuttra’s study showed a 28% drop over three years. But the trials were different, so it’s hard to compare the results directly. Amvuttra is already approved to treat a nerve-related form of ATTR. With this new approval, Alnylam hopes the drug will reach many more people. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) has been approved by the Food and Drug Administration (FDA) to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a potentially fatal disease of the heart muscle. The RNAi therapeutic has been authorised to treat the cardiomyopathy of wild-type or hereditary transthyretin (TTR)-mediated amyloidosis in adults to reduce cardiovascular mortality, hospitalisations and urgent heart failure visits. Approximately 150,000 people in the US are affected by ATTR-CM, in which misfolded TTR protein builds up and causes irreversible cardiovascular damage over time. The majority of patients remain undiagnosed and untreated, Alnylam outlined, adding that a significant portion of those who are treated with commonly used therapies experience disease progression. Given quarterly via a subcutaneous injection, Alnylam’s Amvuttra works with the body’s natural system to rapidly knock down TTR and is already approved in the US to treat the polyneuropathy of hereditary TTR-mediated amyloidosis. The company’s chief executive officer, Yvonne Greenstreet, said: “The FDA approval of Amvuttra for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease.” The FDA’s latest decision on the therapy was based on positive results from the phase 3 HELIOS-B trial, in which Amvuttra demonstrated statistical significance compared to placebo on all ten pre-specified primary and secondary endpoints. In the overall study population, which included patients receiving Amvuttra as a monotherapy and those using the therapy alongside standard treatments, Alnylam’s drug reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular events by 28% during the double-blind treatment period of up to 36 months. In the monotherapy population, Amvuttra reduced the risk of ACM and recurrent cardiovascular events by 33% in the double-blind period and the risk of mortality by 35% at month 42. Mortality in the overall population was also reduced by 36% at month 42. HELIOS-B investigator Ronald Witteles, Stanford University School of Medicine and Stanford Amyloid Center, said: “The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by [Amvuttra’s] ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression. This is a very exciting day for patients with this challenging disease.”