5 Things to Know About the New Cholesterol Drugs

You may have heard about the new cholesterol drugs with the funny name–PCSK9 inhibitors. The FDA approved 2 drugs in this class over the summer, but it took until this week for the nation's top pharmacy benefits manager (PBM), Express Scripts, to agree to pay for them. That coverage starts Monday, so here’s what you need to know:

1.      These drugs are powerful. The 2 drugs approved, Praluent and Repatha, work unlike anything else on the market to lower cholesterol. They affect an enzyme, called the proprotein convertase subtilisin/kexin type 9, or PCSK9. When scientists discovered that people who were naturally missing this enzyme had sharply reduced levels of low-density lipoprotein (LDL) or “bad” cholesterol, they developed medications that could block the enzyme instead. This helps the liver get rid of cholesterol. Studies have shown these medications can cut LDL cholesterol levels by 55% to 60%, well below what is possible with standard treatments.

 

2.      They are expensive. These new drugs are biologics, which puts them in the same realm as treatments for hepatitis C or cancer. When Praluent was approved in July, Sanofi and Regeneron set the wholesale price at $14,600 a year; Amgen followed a month later with an annual price of $14,100 for Repatha. Giant PBMs like Express Scripts and CVS will negotiate steep discounts for their customers, but they still won’t be cheap. And critics note that the drugs will cost much less in Europe.

 

3.      These are not pills; they’re injections. For now, both drugs must be injected every 2 weeks, although a once-a-month version of Repatha is planned. Express Scripts has warned that this will be unfamiliar territory for many patients, who are accustomed to taking a pill a day for cholesterol. (Right now, PCSK9 inhibitors are approved to be used with statins, and some will still take metformin every day.) According to the PBM, “It is critical that all patients who are prescribed these medications receive proper training, close monitoring, and ongoing support.”

 

4.      They’re not for everyone. FDA has not approved these drugs for all patients with high cholesterol. For now, labels call for them to be given to 2 groups: a small number with genetic conditions that create abnormally high cholesterol, called hypercholesterolemia, and a larger group with heart disease; the drugs are limited to those at high-risk for a heart attack or stroke who have been unable to lower their cholestrol with the most powerful statins available. Most US patients with high cholesterol will stay on statins. Unlike European regulators, FDA did not approve these drugs for patients who do not like the side effects of statins.

5.      Some evidence is not in. Both drugs are still being studied for their long-term effects on cardiovascular health and safety, with results expected in 2017. Once FDA has more information, it could expand the number of patients who can get the drugs. By then there will be more competition, since Pfizer is also working on a version.