Amvuttra Solution for injection in pre-filled syringe

USDailyMed:Vutrisiran AU:D N07XX18(WHO) AU:S4(Prescription only)CA:℞-onlyUS:℞-onlyEU:Rx-only 1867157-35-4 DB16699as salt:DBSALT003187 GB4I2JI8UIas salt:28O0WP6Z1P D11917as salt:D11916 Vutrisiran, sold under the brand nameAmvuttra, is amedicationused for the treatment of thepolyneuropathyofhereditary transthyretin-mediated (hATTR) amyloidosisin adults.It is a double stran​​​‍​​‍​​​​‍​​‍‍dedsmall interfering RNA(siRNA; also called RNA interference or RNAi therapeutic) that interferes with the expression of thetransthyretin(TTR) gene.Transthyretin is a serum protein made in the liver whose major function is transport of vitamin A and thyroxine.Rare mutations in the transthyretin gene result in accumulation of large amyloid deposits of misfolded transthyretin molecules most prominently in peripheral nerves and the heart. Patients with hATTR typically present with polyneuropathy or autonomic dysfunction followed by cardiomyopathy which, if untreated, is fatal within 5 to 10 years. Vutrisiran was approved for medical use in the United States in June 2022,in the European Union in September 2022,and in Australia in June 2024.