Amvuttra

Amvuttra is the brand name for vutrisiran, an RNA interference (RNAi) therapy developed by Alnylam Pharmaceuticals. It is approved for adults with hereditary transthyretin-mediated amyloidosis (hATTR) presenting with polyneuropathy, a genetic disorder caused by mutations in the transthyretin (TTR) gene that leads to abnormal amyloid deposition in nerves and other tissues.

Mechanism of action and composition

Amvuttra uses a small interfering RNA (siRNA) that is conjugated with N-acetylgalactosamine (GalNAc) to selectively deliver the siRNA to hepatocytes in the liver. Once inside liver cells, vutrisiran targets TTR mRNA, reducing hepatic production of transthyretin protein and lowering circulating TTR levels. By decreasing TTR synthesis, the therapy aims to slow or modify the progression of nerve damage and related organ involvement in hATTR amyloidosis.

Indication and administration

Amvuttra is indicated for adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy. It is administered by subcutaneous injection, with a dosing schedule typically around every three months, as determined by the treating clinician.

Regulatory status and safety

Regulatory approvals for Amvuttra include the United States and European Union jurisdictions. As with other RNAi therapies, potential adverse events may include injection-site reactions and various systemic symptoms. Clinicians monitor patients for tolerability and any effects on organ systems, and labeling provides guidance on contraindications and precautions.