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

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Additional Relevant Details  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.
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Occurred on:
Jul 28, 2016
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Related Keywords Familial Partial Lipodystrophy, Fpl, Orphan Drug Designation, Volanesorsen