FDAnews
www.fdanews.com/articles/87461-isis-hofh-treatment-granted-orphan-drug-designation

ISIS' HOFH TREATMENT GRANTED ORPHAN DRUG DESIGNATION

June 9, 2006

The FDA has granted orphan drug designation to Isis Pharmaceuticals' ISIS 301012, intended for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder that results in extremely high cholesterol.

ISIS 301012, a second-generation antisense drug, inhibits apoB-100, a protein essential to the synthesis and transport of the type of "bad" cholesterol involved in heart disease -- low density lipoprotein cholesterol (LDL) and very low density lipoprotein (VLDL).

Orphan drug designation entitles Isis to seven years of market exclusivity for ISIS 301012, the company reported.