The FDA approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare inherited disease caused by mutations in the TTR gene.

The agency approval was supported by positive results from a phase 3 clinical trial that enrolled 164 participants. Those who received Amvuttra had significant improvements compared with the placebo group, and half of the treated participants experienced improvements in neuropathy impairment relative to baseline by nine months.

The approved indication for Amvuttra requires an injection treatment in patients once every three months.