Vutrisiran (Amvuttra)

Abstract

CADTH recommends that Amvuttra be reimbursed by public drug plans for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis if certain conditions are met. Amvuttra should only be covered to treat adults with stage 1 or stage 2 genetically confirmed hATTR amyloidosis with polyneuropathy (hATTR-PN) who are symptomatic with early-stage neuropathy, do not have severe heart failure symptoms, and have not had a liver transplant. A patient’s response to treatment with Amvuttra should be assessed at least every 6 months to determine whether they would benefit from continued treatment. Treatment with Amvuttra should not be continued in patients who are permanently bedridden and dependent on assistance for basic activities of daily living or who are receiving end-of-life care. Amvuttra should only be reimbursed if the patient is under the care of a specialist with experience in the diagnosis and management of hATTR-PN and should not be reimbursed if it is used in combination with interfering ribonucleic acid drugs or transthyretin stabilizers. The cost of Amvuttra should be reduced so that it does not cost more than other drugs for hATTR amyloidosis.