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PCSK9 inhibitors: the next cholesterol-busting wonder drugs?

July 08, 2015

Some are calling the potential of a new class of cholesterol-lowering drugs called PCSK9 inhibitors – now in trial at Baystate Medical Center and being debated by an advisory committee of the Food and Drug Administration, who will vote whether to recommend approval – as “one of the biggest developments in a long time in cardiology.”

According to the Centers for Disease Control and Prevention (CDC), nearly a third of all adults – that’s 73 million Americans – suffer from high LDL cholesterol and take extremely popular and highly prescribed statin drugs to lower their levels of bad cholesterol. Cholesterol, a waxy, fat-like substance naturally produced in the body by the liver, is needed by the body, but too much can cause plaque to build up in the arteries, increasing your risk of heart attack and stroke.

A class of biotech drugs called monoclonal antibodies – one developed by Amgen and another by Sanofi and Regeneron Pharmaceuticals – these new drugs block a substance called PCSK9 which interferes with the liver’s ability to remove cholesterol from the blood. They differ from statins, which are taken in pill form, and are instead injected every two to four weeks by patients, similar to the way a diabetic patient takes insulin.

The merits of the new drugs?

According to Dr. Gregory Giugliano, who serves as director of the Cardiac Catheterization Laboratory and of Heart and Vascular Research at Baystate Medical Center, they could offer hope to those who cannot take statins like Lipitor or Crestor because of troubling side effects such as muscle pain, or for those who cannot lower their numbers enough simply by taking statins. This new class of drugs has also been shown to be effective in those patients with an inherited condition called familial hyperlipidemia, which causes very high LDL levels. Also, studies showed the two PCSK9 drugs to reduce bad cholesterol by up to 65 percent, compared to 50 percent by maximum dose statins.

“The PCSK9 inhibitors have been studied alone and in conjunction with statin therapy to combine the effects of both drugs,” said Dr. Giugliano.

At question is whether the PCSK9 inhibitors actually prevent heart attacks or strokes.

“Doctors are awaiting the results of a large multicenter clinical outcomes trial in which some patients at Baystate are enrolled to answer this key question, but the results are about three years away,” said Dr. Giugliano about the FOURIER Trial sponsored by Amgen.

Also to be determined – potential cognitive side effects and others involving kidney problems and higher rates of pancreatitis.

Not to mention the price.

One area the FDA is not allowed to consider in the review of all new drugs is their cost. However, experts predict the new PCSK9 drugs could cost $10,000 per year, compared to a more affordable several hundred dollars per year for statins. Patients taking the new drugs could rack up billions of dollars in new drug spending with the threat of higher premiums.

In other cholesterol-related news, some recent studies suggested statins – among the most popular cholesterol lowering drugs taken by some 25 million Americans – cause memory problems.

However, a new report published in JAMA Internal Medicine says that while statins may contribute to short term memory issues, they tend to resolve over the long term and that these memory problems are not unique to only statins.