Updated 01/20/14: With information on the approval of Juxtapid in Mexico for HoFH.
Aegerion Pharmaceuticals, a Cambridge, Massachusetts biopharmaceutical company focusing on development and commercialization of treatments for rare diseases, launches in the United States in January 2013, orphan drug Juxtapid (Lomitapide). Juxtapid is an oral once-a-day treatment for rare disease Homozygous Familial Hypercholesterolemia (HoFH). HoFH is caused by genetic defects inherited from both parents that affects the function of the LDL receptor, that is responsible for removing bad cholesterol (LDL-C) from the body.
Background Information On Juxtapid For HoFH
• Receives FDA Orphan Drug Designation (ODD) in October 2007
• Launches in US in January 2013
• US price of $235,000 – 295,000/year
• Boxed warning of potential for liver toxicity
• Restricted distribution through Risk Evaluation &…
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