FDA Approves Amvuttra for Heart Conditions

On Monday, March 24, 2025, the U.S. Food and Drug Administration (FDA) granted approval for a groundbreaking medication designed to combat a severe heart condition that impacts thousands of individuals. The newly approved drug, Amvuttra, scientifically known as vutrisiran, is produced by Alnylam Pharmaceuticals and targets transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is characterized by the accumulation of harmful proteins in the heart, which impairs its ability to effectively pump blood, as explained by the American Heart Association (AHA).

Clinical trials have demonstrated that Amvuttra significantly reduces the risk of mortality and heart-related issues by 28% over a three-year period when compared to a placebo. The medication is administered via injection every three months. Alnylam’s CEO, Yvonne Greenstreet, expressed optimism in a conversation with STAT News, highlighting the drug's potential to become a flagship franchise for the company and its promise to benefit numerous patients. Greenstreet emphasized the transformational impact of this approval for Alnylam Pharmaceuticals.

Despite its promising benefits, the treatment is accompanied by a substantial financial burden. Each dose of Amvuttra is priced at approximately $119,000, resulting in an annual cost of $476,000, which exceeds the pricing of comparable treatments from BridgeBio and Pfizer for the same condition. Dr. Mani Foroohar, an analyst with Leerink, a healthcare investment firm, noted the large, underdiagnosed market and significant unmet needs within the ATTR-CM patient community.

However, the high price tag poses challenges in terms of accessibility, particularly for patients reliant on Medicare Advantage plans. Private insurers may opt for more affordable alternatives, such as oral medications offered by Pfizer or BridgeBio. Unlike these treatments, Amvuttra employs RNA technology to inhibit the production of the harmful protein, whereas other drugs aim to stabilize the protein before it can inflict damage.

Clinical trials have revealed that BridgeBio’s medication achieved a 42% reduction in mortality and cardiac hospitalizations over a 30-month period. Amvuttra’s trial indicated a 28% reduction over three years, although the differing methodologies make direct comparison of results challenging.

Amvuttra is already approved for treating a nerve-related variation of ATTR. With the latest FDA approval, Alnylam Pharmaceuticals seeks to extend the drug's reach to a broader demographic of patients suffering from ATTR-CM.