
Alnylam (Nasdaq: ALNY) has secured expanded UK regulatory approval for Amvuttra (vutrisiran), broadening its indication to include treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in adults.
ATTR-CM is a progressive and ultimately life-shortening condition caused by the buildup of misfolded transthyretin proteins in cardiac tissue. This can lead to worsening heart failure and other severe complications.
The Medicines and Healthcare products Regulatory Agency granted the green light for use in both wild-type and hereditary forms of the disease, which affects the heart. The treatment works by silencing the gene responsible for producing the TTR protein, which is central to the disease pathology.
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