FDA approves new orphan drug for genetic cholesterol disorder

Hannah Blake


The FDA has approved a new drug that not only treats a rare genetic disease that causes extremely high cholesterol levels, but does so using first-of-its-kind technology that has been enhanced over two decades.

Developed by Sanofi subsidiary, Genzyme, in partnership with Isis Pharmaceuticals, Kynamro (mipomersen sodium) is to be given as a 200 mg weekly subcutaneous injection. It’s been approved as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (Apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

The FDA approval triggers a US $25 million milestone payment to Isis from Genzyme.

“Today’s FDA approval of KYNAMRO is great news for patients with HoFH who are in need of additional treatment options for this rare, and often under-diagnosed disease. As the leader in treatments for rare diseases, we are pleased to bring our expertise to HoFH patients living with this serious condition to better help them manage their disease.”

Genzyme President and CEO David Meeker, M.D.

HoFH is a genetic condition that affects about one out of every one million people in the United States, which means it is classed as a rare disease. The condition 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.

In December 2012, the FDA approved Aegerion Pharmaceuticals’ Juxtapid (lomitapide) to reduce LDL-C, total cholesterol, apolipoprotein B, and non HDL-C in patients with HoFH.


Related news:

F.D.A. Approves Genetic Drug to Treat Rare Disease (New York Times)

Reference links:

Genzyme press release

FDA press release


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