Alnylam Pharmaceuticals (ALNY) has submitted a Type II Variation to the European Medicines Agency concerning vutrisiran, an investigational RNA interference therapeutic currently under development for treating ATTR amyloidosis with cardiomyopathy. Vutrisiran, the generic form of AMVUTTRA, has already received approval in the European Union for addressing hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
The regulatory submission is backed by encouraging outcomes from the HELIOS-B Phase 3 trial, which was a randomized, double-blind, placebo-controlled multicenter study conducted globally. Additionally, a supplemental New Drug Application (sNDA) for vutrisiran has been filed with the U.S. Food and Drug Administration (FDA) for the treatment of ATTR-CM.
