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FDA Considers New Cholesterol-Lowering Drugs

Advisers to the FDA will meet this week to consider approval of a new class of cholesterol drugs that could help hundreds of thousands of people.

Advisers to the Food and Drug Administration will meet this week to consider approval of a new class of cholesterol-lowering drugs that could help hundreds of thousands of people who cannot take statins.

They’re injectable, so they wouldn’t be as easy to take as a pill. And they are in a class of biotech drugs called monoclonal antibodies, so they are likely to be very pricey – perhaps $10,000 a year. But studies show they can lower bad cholesterol to extremely low levels, without the side-effects suffered by some people who take statins. Heart experts are very excited about them.

“These drugs lower bad cholesterol more than any drugs we've ever had in history,” Dr. Steven Nissen, a cardiologist at the Cleveland Clinic, told NBC News.

“These drugs lower bad cholesterol more than any drugs we've ever had in history."

“The problem with statin drugs is that they can't get everybody's LDL down. Some people can't take them due to side effects,” Nissen added.

“And a lot of people out there that we really want to get lower, we had no way to get them there until these drugs came along.”

Statins, which include brand names such as Lipitor, Mevacor, Crestor and Zocor — are extremely popular. They’re prescribed to about a 15 percent of U.S. adults and range in cost from about $11 for the cheapest generic version to $200 for a pricey name-brand. They are among the most commonly prescribed drugs in the world.

But they have side-effects, including a rare type of muscle breakdown and weakness that affects somewhere between 5 and 15 percent of patients who take them, depending on who’s doing the estimating.

Marquerite Echols McCoy was one of them.

"Every time they put me on a statin my legs cramped severely," McCoy told NBC News.

In 2012, the FDA updated labeling on statins to include warnings about confusion and memory loss, elevated blood sugar leading to Type 2 diabetes, and muscle weakness.

The new drugs -- Sanofi’s alirocumab and Amgen’s evolocumab -- work in a completely different way from statins. They interfere with an enzyme on liver cells called PCSK9. When people have too much PCSK9, it seems to interfere with the liver’s ability to pull low density lipoprotein, the LDL or bad cholesterol, out of the blood. The new drugs are made of lab-engineered antibodies that interfere with PCSK9 and allow the liver to do its LDL-lowering job better.

“When PCSK9 levels are low or the function of PCSK9 is inhibited, you have more LDL receptors, and the LDL levels in the blood will drop. The goal is to have a lower LDL level in the blood,” said Dr. Elliott Antman, of Brigham and Women’s Hospital and Harvard Medical School, who is president of the American Heart Association.

“It was discovered that individuals born with very low levels of PCSK9 or low levels of a functioning form of PCSK9 have low LDL levels and almost never get vascular disease.”

McCoy tried one as part of a clinical trial. "I give myself three injections in my thigh or stomach once a month," said McCoy, 60, who lives in Toledo, Ohio.

"It’s not painful. I do it myself. I have no side effects. It’s an easier way for me to get rid of the bad cholesterol. No matter what exercise I did, my cholesterol was high. Now, my cholesterol is down in mid 70s and my HDL (high density lipoprotein or 'good' cholesterol) is where it’s supposed to be. I’m having no cramps, and I feel great."

One of the first approvals is likely to be for people who have an inherited form of high cholesterol, called familial hypercholesterolemia, Antman said. They can eat a very healthful diet and still have super-high cholesterol.

“You can give this preparation by an injection under the skin every two weeks or every four weeks, depending upon the preparation and it will inhibit PSCK9 activity and dramatically lower LDL cholesterol – reductions can be greater than 50 percent,” Antman said.

That means the LDL doesn’t build up on artery walls, causing heart disease and causing blockages that can starve the heart of blood or, worse, break off and cause a heart attack or stroke.

"Some people think they will have enormous impact, including me. We may find we’re halting disease in its tracks," Nissen said.

"We may find we’re halting disease in its tracks."

The FDA’s advisory committee will examine Sanofi’s drug Tuesday and Amgen’s drug Wednesday

The American Heart Association and American College of Cardiology have tweaked cholesterol guidelines and say people should just take the best possible dose of statins. Previous guidelines focused on numbers. People were supposed to aim for a total cholesterol level of less than 200, keeping LDL to 130 for average people and under 100 for those considered at risk of a heart attack. People at the highest risk, like heart attack survivors, were supposed to keep LDL to 70 or below.

The new drugs have been developed quickly, said Nissen, who helped test them for the makers.

“These drugs have gone from the bench to the bedside faster than almost any drugs in history. It's really a remarkable story,” he said. “They don't seem to cause muscle side effects that statins have, which is one of their principal advantages.”