FDA approves Tegsedi to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis. Akcea Therapeutics + Ionis Pharma

Akcea Therapeutics, Inc.an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. announced that the FDA has approved Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Tegsedi is now approved in the U.S., European Union and Canada.

�Tegsedi is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis. We believe this profile will make Tefsedi an excellent choice for many patients and that it�s self-administration gives the flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed,� said Paula Soteropoulos, chief executive officer at Akcea Therapeutics.

In hATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. Tegsedi targets the disease at its source by reducing the production of TTR protein. In the NEURO-TTR study, treatment with TEGSEDI produced up to a 79% mean decrease from baseline in serum TTR protein in patients regardless of TTR mutation, sex, age, or race. The FDA�s approval of TEGSEDI was based on results from the Phase III NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with Tegsedi 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.

TegsediI is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified risks.