Pipeline Update: Lipid Disorders

Dec 17, 2014
PCSK9 inhibitors to treat high cholesterol are expected to have a significant impact on drug spend.
  • High Blood Cholesterol

Familial hypercholesterolemia (FH) is an inherited condition that results in the body not being able to remove low-density lipoprotein (LDL) cholesterol – bad cholesterol – from circulation. This causes patients to develop cardiovascular disease early in life.

The disease is inherited via a defective gene and patients with two defective gene copies have a more severe version of the disease, also known as homozygous familial hypercholesterolemia (HoFH). This is a rare condition affecting approximately 3,000 Americans. Those with one copy have heterozygous familial hypercholesterolemia (HeFH), which affects as many as one million Americans.

Patients with FH are frequently treated with statins and other cholesterol-lowering agents. Some patients also require apheresis, a mechanical procedure that removes LDL cholesterol from the blood. In addition, Kynamro® (mipomersen) and Juxtapid® (lomitapide) are medications that have been available since 2013 to treat HoFH. Even with the availability of current treatment options for familial hypercholesterolemia, many patients still have elevated LDL cholesterol.

New biologic drugs that lower cholesterol through inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9 inhibitors) are currently in development for high cholesterol in patients with familial hypercholesterolemia as well as those who have had an inadequate response or intolerance to statin therapy. The new drugs are expected to be approved starting in mid-2015.

PCSK9 inhibitors are expected to have a significant impact on drug spend. While initially, use may be primarily reserved for patients with familial hypercholesterolemia and those intolerant to statins, additional data on the impact for cardiovascular use is expected in 2017.

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