Akcea Therapeutics Receives Orphan Designation in Europe for Volanesorsen for the Treatment of Familial Partial Lipodystrophy (FPL)

CAMBRIDGE, Mass., July 28, 2016 /PRNewswire/ -- Akcea Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the European Commission (EC) has designated volanesorsen as an orphan medicinal product for the treatment of familial partial lipodystrophy (FPL). FPL is a rare lipid disorder characterized by abnormal fat distribution across the body and a range of metabolic abnormalities, including severe type 2 diabetes, high triglycerides, and accumulation of fat in the liver. The EC's approval follows a positive opinion in June from the European Medicine Agency's (EMA) Committee for Orphan Medicinal Products. Volanesorsen is being developed for the treatment of two rare, genetic cardiometabolic diseases: familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy.

The release as fetched from its publisher. ionis.com ↗