Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis

A Phase III study randomly assigned individuals with hereditary transthyretin amyloidosis with polyneuropathy to receive I.V. patisiran or placebo once every 3 weeks. Current treatment options for the disease are limited. However, the study found that patisiran, an RNAi therapeutic, significantly improved neuropathy in recipients.

A Phase III study randomly assigned individuals with hereditary transthyretin amyloidosis with polyneuropathy to receive I.V. patisiran or placebo once every 3 weeks. Current treatment options for the disease are limited. However, the study found that patisiran, an RNAi therapeutic, significantly improved neuropathy in recipients. "The effects extended across the sensorimotor and autonomic domains and were consistent across patient subgroups," the researchers report, noting that treatment also resulted in improvements in quality of life, walking, nutritional status, and other factors. There were mild-to-moderate infusion-related reactions, though the incidence of them decline over time, while the number of deaths overall as well as in a cardiac subgroup were similar between the two treatment groups. "In conclusion," the researcher write, "the APOLLO Phase III trial showed that patisiran provided benefit to patients with hereditary transthyretin amyloidosis by treating a broad range of symptoms."