{"title":"FDA approves Alnylam’s anti-amyloid RNAi drug for cardiomyopathy","authors":"","doi":"10.1038/s41587-025-02656-2","DOIUrl":null,"url":null,"abstract":"<p>The first RNA interference (RNAi) drug for treating heart failure caused by amyloidosis was approved by the US Food and Drug Administration (FDA) in March. Alnylam has the go-ahead for Amvuttra (vutrisiran) to treat transthyretin amyloid cardiomyopathy (ATTR-CM) caused by sporadic or hereditary ATTR, expanding the drug’s label from a previous nod in 2022 for polyneuropathy caused by transthyretin amyloidosis.</p><p>The disease ATTR-CM is rapidly progressive and fatal, and is caused by misfolding of the liver transport protein transthyretin. The abnormal protein fibrils are prone to aggregate and form damaging deposit in many organs, including the heart, leading to cardiovascular death, hospitalizations and urgent heart failure visits. Estimates suggest ATTR-CM affects over 300,000 people globally, but most cases are thought to be undiagnosed, and current treatments cannot halt its progression.</p>","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 1","pages":""},"PeriodicalIF":33.1000,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nature biotechnology","FirstCategoryId":"5","ListUrlMain":"https://doi.org/10.1038/s41587-025-02656-2","RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
The first RNA interference (RNAi) drug for treating heart failure caused by amyloidosis was approved by the US Food and Drug Administration (FDA) in March. Alnylam has the go-ahead for Amvuttra (vutrisiran) to treat transthyretin amyloid cardiomyopathy (ATTR-CM) caused by sporadic or hereditary ATTR, expanding the drug’s label from a previous nod in 2022 for polyneuropathy caused by transthyretin amyloidosis.
The disease ATTR-CM is rapidly progressive and fatal, and is caused by misfolding of the liver transport protein transthyretin. The abnormal protein fibrils are prone to aggregate and form damaging deposit in many organs, including the heart, leading to cardiovascular death, hospitalizations and urgent heart failure visits. Estimates suggest ATTR-CM affects over 300,000 people globally, but most cases are thought to be undiagnosed, and current treatments cannot halt its progression.
期刊介绍：
Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research.
The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field.
Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology.
In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.
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