FDA grants 'orphan drug' status to Aegerion drug
CAMBRIDGE -- Aegerion Pharmaceuticals has received federal orphan drug status for its lead candidate, a treatment for people with severe lipid disorders.
Orphan drug status is given to drugs that could treat rare diseases or conditions that affect fewer than 200,000 people in the United States. The approval, announced today, comes with incentives like seven years of marketing exclusivity following approval and tax credits, among other benefits.
Aegerion's once-a-day drug candidate lomitapide is used to treat familial chylomicronemia, a rare genetic disorder that leads to extremely high levels of triglycerides in the blood.
The drug, which is in late-stage clinical testing, prevents the liver and intestines from secreting lipids into the bloodstream.
The approval by the Food and Drug Administration follows approval in Europe, where the drug can be used to treat familial chylomicronemia.
Shares of Aegerion Pharmaceuticals Inc. climbed 2 cents to $15.14 in afternoon trading.