Alnylam's next-gen ATTR amyloidosis drug hits goals in phase 3, teeing up approval filing

A phase 3 clinical trial of Alnylam’s subcutaneous, long-acting treatment for transthyretin-mediated (ATTR) amyloidosis has met its primary endpoint. The success sets the RNAi specialist up to file for FDA approval early this year, although a lack of data comparing the experimental vutrisiran to Alnylam’s own ATTR incumbent Onpattro make it impossible to tell just how good the results are.