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Aegerion gains orphan drug status for lipid disorder drug

Wed, 03/16/2011 - 10:35am
Mass High Tech: The Journal of New England Technology

Aegerion Pharmaceuticals Inc., a lipid disorder treatment firm in Cambridge, has been granted orphan drug designation from the U.S. Food and Drug Administration for its treatment of familial chylomicronemia (FC), a rare genetic disorder.

The drug candidate, lomitapide, is currently in Phase 3 clinical trials to treat Homozygous Familial Hypercholesterolemia (HoFH), a disorder marked by high levels of low-density lipoprotein in the blood. Lomitapide already has orphan drug designation in the U.S. for HoFH.

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