Genzyme, a Sanofi company (SNY), and Isis Pharmaceuticals Inc. (ISIS), announced that the U.S. Food and Drug Administration or FDA has approved its New Drug Application or NDA for Kynamro (mipomersen sodium) injection.
Kynamro, given as a 200 mg weekly subcutaneous injection, has been approved as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol or LDL-C, apolipoprotein B (Apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia or HoFH.
HoFH is a rare inherited condition that makes the body unable to remove LDL cholesterol, often called the "bad" cholesterol, from the blood, causing abnormally high levels of circulating LDL cholesterol.
In the United States, HoFH, an orphan indication, occurs in approximately one in one million individuals. For those with HoFH, heart attacks and death often occur before age 30.
The FDA approval triggers a $25 million milestone payment to Isis from Genzyme.
Kynamro contains a Boxed Warning citing the risk of hepatic toxicity. Patients taking Kynamro should have liver enzyme testing before starting the drug and periodically thereafter.
The company said that Kynamro is not recommended in patients with severe renal impairment, clinically significant proteinuria, or on renal dialysis.
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