Vutrisiran recommended for EU approval by CHMP for the treatment of ATTR-CM
The RNAi therapeutic vutrisiran has received a positive opinion by the CHMP of the EMA for the treatment of adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion recommending the approval of vutrisiran for the treatment of wild-type or hereditary transthyretin amyloidosis in adults with cardiomyopathy (ATTR-CM). This recommendation by the CHMP is based on the results of the HELIOS-B study.
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, treatment with vutrisiran reduced the risk of the primary composite outcome of all-cause mortality or recurrent CV events in patients with ATTR-CM, compared with placebo. All pre-specified secondary endpoints were also met. The treatment effects of vutrisiran were consistent across all patient subgroups, including in patients on concomitant TTR stabilizer.
Vutrisiran is an RNAi therapeutic that targets hepatic TTR production. Currently, vutrisiran is approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy (ATTRv-PN).
The decision by the European Commission is expected in June 2025.