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Heart & metabolic

Vutrisiran — ATTR amyloidosis

A quarterly injection that reduces the protein driving ATTR amyloidosis.

ATTR amyloidosis is caused by a misfolded transthyretin protein depositing in nerves and heart muscle. Vutrisiran is a small interfering RNA that reduces transthyretin production at the source, given as a subcutaneous injection every three months.

For patients in markets where the product is not yet registered, the zone’s special-access pathway can make it reachable well before a national approval. Diagnosis must be confirmed by genetic testing and, where relevant, cardiac imaging.

What it does not do

  • Confirming the diagnosis — and the genotype — comes first. This is not a treatment for other forms of amyloidosis.
  • It slows progression; it does not reverse existing nerve or heart damage.
  • Patients already stable on an alternative therapy should discuss switching with their own physician before travelling.

These limits are written by us, not by the hospital. If a provider presents this therapy without any of them, treat that as a warning sign.

IndicationHereditary ATTR amyloidosis with polyneuropathy or cardiomyopathy
Approved inUnited States, European Union, Japan
Status in ChinaFirst clinical use in China delivered in the zone
Assessment requiredYes — physician assessment first
Delivered at2 hospital(s)
Verified 2026-10
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Availability moves with batches and indications. A two-line enquiry is enough for us to confirm whether it is reachable this quarter.