Inotersen to address polyneuropathyDecember 13, 2018
The US Food and Drug Administration has approved inotersen (Tegsedi) for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Inotersenis an RNA-targeting therapeutic that reduces the production of TTR protein through a once-weekly subcutaneous injection offering a treatment for people living with polyneuropathy caused by hATTR amyloidosis.
In hATTRamyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. Inotersen targets the disease at its source by reducing the production of TTR protein.
The FDA looked at results from the Phase 3 NEURO-TTR study in patients with hATTRamyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with inotersen experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, Akcea Therapeutics, Inc and Ionis Pharmaceuticals, Inc, announced.