- News in Brief
- Published:
volume 43, page 457 (2025)Cite this article
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.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
24,99 € / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
195,33 € per year
only 16,28 € per issue
Buy this article
- Purchase on SpringerLink
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Rights and permissions
About this article
Cite this article
FDA approves Alnylam’s anti-amyloid RNAi drug for cardiomyopathy.
Nat Biotechnol 43, 457 (2025). https://doi.org/10.1038/s41587-025-02656-2
-
Published:
-
Issue Date:
-
DOI: https://doi.org/10.1038/s41587-025-02656-2
