FDA Approves AMVUTTRA for ATTR CM Treatment

Published: 24 Mar 2025

Author: Precedence Research

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Alnylam Pharmaceuticals announced that it has received approval from the FDA for its supplement to the new drug application for AMVUTTRA® (vutrisiran) as an extension of treatment for cardiomyopathy due to wild-type or inherited transthyretin-mediated amyloidosis, adult only.

The drug is a new treatment for ATTR-CM as it covers cardiovascular mortality, hospitalization, and urgent heart failure visits. ATTR-CM is a progressing, completely fatal condition of about 150,000 people in the U.S. and a much wider swath-300,000 plus worldwide. Currently, no cures exist for this malady. Many go undiagnosed or progress despite treatment.  

A New Era for ATTR-CM Treatment

AMVUTTRA® (vutrisiran) is now FDA-approved via supplemental New Drug Application, thus broadening treatment for cardiomyopathy due to wild-type or inherited transthyretin-mediated amyloidosis in adults. As a breakthrough therapy, it is directed at cardiovascular mortality, hospitalizations, and urgent heart failure visits, addressing an ailment affecting over 300,000 individuals worldwide. Currently, there is no cure for ATTR-CM; hence, many patients remain undiagnosed, while others progress despite treatment. 

Clinical Trial Evidence: HELIOS-B Study 

The approval is based on the outcome of the HELIOS-B Phase 3 clinical trial. The study reported: a 28% reduction in all-cause mortality and recurrent cardiovascular events across a 36-month double-blind period; a 42-month mortality reduction of 36%; and a 33% reduction in ACM and CV events in the monotherapy population. The involved patients also showed improvements in functional capacity and quality of life, as well as improvement in biomarkers NT-proBNP and troponin I. The HELIOS-B study confirmed the safety profile of AMVUTTRA: the most common adverse effects were pain in the extremities, joint pain, dyspnea, and decreased levels of vitamin A. 

Expert Opinions and Patient Impact  

The FDA has approved AMVUTTRA, a rare drug for treating Alzheimer's disease, while Ronald Witteles, M.D., a HELIOS-B investigator and professor at Stanford University School of Medicine, said. The trial enrolled patients from real-world demographics, demonstrating that the drug can prolong survival, diminish hospitalization, and improve patient well-being. AMVUTTRA has been welcomed by patient advocacy groups like the Amyloidosis Support Groups as a ray of hope for the afflicted. Alnylam has been committed to ensuring broad access to AMVUTTRA, with insurance coverage for about 99 percent of hATTR-PN patients and similar expectations for ATTR-CM. They also provide Alnylam Assist®, which helps with insurance coverage assistance, offers first doses free, and provides educational resources for patients and healthcare providers. 

Global Regulatory Expansion

After obtaining approval from the U.S. FDA, Alnylam is seeking regulatory approvals for its RNAi therapeutic AMVUTTRA in various international markets. The European Medicines Agency, Brazil's ANVISA, and Japan's PMDA are currently reviewing the applications. Further submissions are planned in 2025 to increase patient access to AMVUTTRA worldwide. AMVUTTRA is the first RNAi therapeutic approved for ATTR-CM and hATTR-PN and demonstrates the future potential of RNAi technology in solving medical issues. Alnylam continues to pursue its mission of improving patient outcomes and quality of life.