EC approval for vutrisiran for the treatment of ATTR-CM
The RNAi therapeutic vutrisiran has been approved in the EU for the treatment of adults with wild-type or hereditary transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
The approval broadens the indication of vutrisiran, which already had an indication for the treatment of the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. This decision by the EMA is based on positive results from HELIOS-B.
HELIOS-B was a global, multicenter, placebo-controlled, double-blind, phase 3 RCT in which 655 patients with ATTR-CM were randomized to subcutaneous vutrisiran once every three months or placebo. In HELIOS-B, vutrisiran met all 10 pre-specified primary and secondary endpoints. Vutrisiran reduced the primary composite endpoint of all-cause mortality or recurrent cardiovascular events by 28% compared with placebo in HELIOS-B.
HELIOS-B enrolled a diverse population of ATTR-CM patients, including those receiving substantial concurrent use of available standard-of-care therapies such as the TTR stabilizer tafamidis and SGLT2 inhibitors. The treatment effects of vutrisiran were consistent across the overall and monotherapy populations.
Vutrisiran is an siRNA that inhibits the production of TTR protein.