FDA green light for hATTR treatment Amvuttra

RNAi therapeutics company Alnylam Pharmaceuticals (Nasdaq: ALNY) today 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.

The approval comes a month earlier than the expected FDA decision date of July 14, and the news drove Alnylam’s shares up nearly 7% to $136.04 in early trading

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 nine-month results from the HELIOS-A Phase III study, where Amvuttra significantly improved the signs and symptoms of polyneuropathy, with more than 50% of patients experiencing halting or reversal of their disease manifestations.