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The Food and Drug Administration of the United States gave its approval for the drug Kynamro to start treating patients with a rare genetic disorder.

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder where the patient has high blood cholesterol. Kynamro (mipomersen sodium) has been approved to be administered along with the lipid-lowering medication and diet which is already being prescribed to patients. Kynamro has been found effective in lowering the levels of low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol (non HDL-C).

Almost one in a million people in the U.S. suffers from HoFH, where the body's mechanism to remove LDL-C is impaired, which increases the level of LDL-C in the blood. Heart attacks and death before the age of 30 is a common occurrence for people suffering from HoFH. Kynamro is an 'orphan drug approval', a term commonly used when a medicine is designed to treat a disease which affects less than 200,000 people.

The drug underwent a clinical trial involving 51 patients with HoFH to test its safety and efficacy. There was a marked reduction of LDL-C in the blood by 25 percent in the first 26 weeks. However, excessive use of Kynamro can lead to liver enzyme abnormalities, excessive fat in the liver and liver disease, a warning which is already given in its medicine boxes.

"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." Eric Colman, M.D., deputy director of the Division of Metabolism and Endocrinology Products in the FDA's Center for Drug Evaluation and Research, said in an agency news release

A Risk Evaluation and Mitigation Strategy (REMS) is applied while approving Kynamro, which ensures the safe use of the drug. This means that the drug will require the certification of both the doctor prescribing it and the pharmacy selling it. The safe-use conditions are documented and a prescription authorization form needs to be filled for each new prescription.

The common side-effects of the injection are reactions at the site of injection, flu-like symptoms, nausea, headache and elevation in the liver enzymes.