www.fdanews.com/articles/87283-isis-hofh-treatment-gets-orphan-drug-designation
ISIS' HOFH TREATMENT GETS ORPHAN DRUG DESIGNATION
June 2, 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).