Vutrisiran

How it works

It is an RNA-interference agent that reduces hepatic production of both mutant and wild-type transthyretin, lowering the protein available to form amyloid deposits.

Prescribing in practice

• Because transthyretin transports retinol, treatment lowers serum vitamin A, so vitamin A supplementation at the recommended daily allowance is advised and ophthalmic symptoms should be assessed.
• It is given as a regular subcutaneous injection under specialist neurology or amyloidosis supervision.
• Injection-site reactions can occur but are generally mild and transient.

Monitoring

Monitor neurological status and disease progression, and assess for symptoms of vitamin A deficiency such as visual disturbance.

Counselling the patient

• Take vitamin A supplementation as advised and report any problems with night vision or dry eyes.
• Injection-site reactions are usually mild and settle on their own.
• Attend regular review appointments to monitor your condition.

Evidence & guidelines

Vutrisiran is supported by the HELIOS-A trial and is licensed for hereditary transthyretin amyloidosis with polyneuropathy.