ISIS' HOFH TREATMENT GETS ORPHAN DRUG DESIGNATION

A A

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).