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FDA Approves Second Treatment for Hereditary Amyloidosis (hATTR)

FDA Approves Second Treatment for Hereditary Amyloidosis (hATTR)

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (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 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.

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.

Read the entire press release here.