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Amvuttra (vutrisiran)
Australian Prescription Medicine Decision Summary
Published
Product name
Amvuttra
Active ingredient
Vutrisiran
Submission type
Type A (New chemical entity)
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Amvuttra was approved for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
How this medicine works
Amvuttra is used to treat polyneuropathy (when multiple peripheral nerves are damaged) caused by hereditary transthyretin-mediated (hATTR) amyloidosis. In hATTR, abnormal proteins called amyloids accumulate around the nerves. Amvuttra contains vutrisiran, a small interfering ribonucleic acid (siRNA) that is delivered to liver cells and specifically targets transthyretin (TTR). Through a natural process called RNA interference (RNAi), vutrisiran causes the degradation of TTR mRNA in the liver, resulting in the reduction of TTR protein levels.
TTR transports vitamin A (retinol) and a hormone called thyroxine throughout the body. To perform this transport function, TTR units must form a tetramer (a protein complex made up of four TTR subunits). Patients with hATTR possess TTR gene variants that prevent tetramer formation and form fibril clumps instead which form amyloid. These amyloid deposits cause the symptoms of hATTR by accumulating in certain tissues. Amvuttra alleviates these symptoms by reducing TTR synthesis which leads to reduced levels of amyloid deposits.
Why the TGA approved or did not approve this medicine
The decision to approve Amvuttra was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology studies conducted in animal models), clinical (pharmacology, safety and efficacy trials in humans) and risk management plan data submitted to the TGA by the sponsor.
Key trials in humans that supported the proposed indication included a Phase 1 single-dose study in healthy volunteers and a pivotal Phase 3 study in hATTR amyloidosis patients with polyneuropathy.
Consumer Medicine Information (CMI)
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
The Australian CMIs can be accessed through the ARTG details hyperlink below.
Product Information (PI)
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
The Australian PIs can be accessed through the ARTG details hyperlink below.
Sponsor
Other resources
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For information on medicines subsidised by the Australian Government refer to the Pharmaceutical Benefits Scheme (PBS) website.
For data and reports on health and welfare topics in Australia refer to the Australian Institute of Health and Welfare website.