US food and drug administration approves orphan drug to treat rare cholesterol disorder
The US Food and Drug Administration approved Kynamro (mipomersen sodium) injection as an addition to lipid-lowering medications and diet to treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).
The addition of Kynamro helps to reduce low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol (non HDL-C).
HoFH, an inherited condition that affects about one out of every one million people in US, occurs when the body is unable to remove LDL-C, often called "bad" cholesterol, from the blood causing abnormally high levels of circulating LDL-C. For those with HoFH, heart attacks and death often occur before age 30.
Kynamro is an orphan drug approval, meaning it was developed to treat a disorder affecting fewer than 200,000 people. In December last year, the FDA approved Juxtapid (lomitapide) to reduce LDL-C, total cholesterol, apolipoprotein B, and non HDL-C in patients with HoFH.
"Kynamro, an injection given once a week, works with other lipid-lowering medications and diet to impair the creation of the lipid particles that ultimately give rise to LDL-C," said Eric Colman deputy director of metabolism and endocrinology products at FDA's Center for Drug Evaluation and Research.
The safety and effectiveness of Kynamro were evaluated in a clinical trial of 51 patients with HoFH. On average, levels of LDL-C fell by about 25% during the first 26 weeks in those receiving the drug.
Kynamro carries a boxed warning on the serious risk of liver toxicity because it is associated with liver enzyme abnormalities and accumulation of fat in the liver, which could lead to progressive liver disease with chronic use.