The disease affects an estimated 150,000 people in the U.S.
of ConsumerAffairsHeart disease patients now have another treatment option. The U.S. Food and Drug Administration has approved Alnylam Pharmaceuticals treatment for a progressive heart condition, vutrisiran, marketed under the brand name Amvuttra.
The drug will be an approved treatment for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. Amvuttra becomes the first and only therapy authorized by the FDA to treat both ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis, according to the company.
ATTR-CM, a progressive and fatal disease affecting an estimated 150,000 people in the U.S. and over 300,000 worldwide, involves the buildup of protein, leading to irreversible heart damage and premature death. Until now, treatment options have been limited, and many patients experience disease progression despite therapy.
Clinically differentiated treatment option
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, said Yvonne Greenstreet, CEO of Alnylam, in a press release announcing the approval.
According to the company, the treatment works by targeting the source of the disease, rapidly reducing TTR protein production with only four subcutaneous doses per year. This reduction in TTR deposition has been shown to significantly decrease cardiovascular damage and improve patient outcomes, the company said.
Other drugs to treat this heart condition are produced by BridgeBio and Pfizer.
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Posted: 2025-03-21 13:51:38
