Inotersen treatment for patients with hereditary transthyretin amyloidosis

Inotersen, which inhibits hepatic production of transthyretin, may be a viable intervention for hereditary transthyretin amyloidosis. As part of a Phase III clinical trial, researchers randomized 112 patients with stage 1 or stage 2 disease and polyneuropathy to receive subcutaneous injections of inotersen once per week.

Inotersen, which inhibits hepatic production of transthyretin, may be a viable intervention for hereditary transthyretin amyloidosis. As part of a Phase III clinical trial, researchers randomized 112 patients with stage 1 or stage 2 disease and polyneuropathy to receive subcutaneous injections of inotersen once per week. Another 60 participants were assigned to placebo. After 15 months of treatment, neuropathy impairment and quality of life scores with significantly improved in the inotersen group. The effect was independent of disease progression, mutation type, or the presence or absence of cardiomyopathy. The primary serious adverse events among the inotersen patients were thrombocytopenia and glomerulonephritis, which were subsequently managed via enhanced monitoring.