Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced that the US Food and Drug Administration (FDA) approved Amvuttra (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. The FDA approval is based on positive 9-month results from the HELIOS-A phase 3 study, where Amvuttra significantly improved the signs and symptoms of polyneuropathy, with more than 50 per cent of patients experiencing halting or reversal of their disease manifestations.
