By Matt Sloane
WebMD Health News
Reviewed by Brunilda Nazario, MD
June 10, 2015 -- A new class of medications can lower "bad" LDL cholesterol to unheard-of levels, and it may be on the horizon.
The new class is called PCSK9 inhibitors. An FDA advisory panel on Tuesday recommended that the agency approve the first drug, called alirocumab (Praluent). On Wednesday, the panel recommended approval for a second medication, evolocumab (Repatha).
The FDA typically follows the recommendation of its advisory panels. The agency will decide whether to approve both drugs later this summer.
PCSK9 drugs have worked particularly well for people with a family history of high cholesterol and those who may not be able to tolerate widely-prescribed statin drugs. But their estimated price tag is steep.
We asked two experts for their opinions on these new medications.
How are PCSK9 inhibitors different than statins?
"Statins work by reducing the production of cholesterol in the liver," says Melina Jampolis, MD, a physician nutrition specialist in California. "These drugs work by pulling [cholesterol] out of the bloodstream more effectively."
More specifically, these drugs allow the molecules that sweep up your bad cholesterol to keep working longer and harder than they would normally.
Who'd benefit from these meds?
Up to 20% of people on statins stop taking them "because they complain of muscle aches, memory loss, and flu-like symptoms, and we see changes in their liver function," Jampolis says. That means PCSK9 inhibitors might be ideal for them.
Also, some people with genetically high cholesterol are unable to get their numbers down with statins alone, she says. They face an incredibly high risk of heart disease and might benefit greatly from these drugs, she says.
With this new class of medications, "one can get cholesterol levels down to what we're born with, so never-before-seen low levels," says Christopher Cannon, MD, a professor of cardiology at Harvard Medical School. He was involved with the research into these drugs.
Also, they can be used in addition to statins to boost how much someone benefits.
"They can reduce cholesterol, and the bad cholesterol in particular, by 50% more, and that's on top of statin medications," Cannon says.
How do you take the drugs?
Unlike statins, which come in pill form, PCSK9-inhibitors come as a shot only. You have to give yourself a shot under the skin, Jampolis says.
The good news, she says, is that it only needs to be done every 2-4 weeks. That may be a plus for people who often forget to take their statin pills, Cannon says.
"Many of us have thought, 'Well, who would want to take an injection when you could take a pill?'" he says. "On the other hand, if you do that every 2 weeks, or sometimes every 4 weeks, maybe that's easier, and you don't have to worry about taking your pills every day."
Are there side effects?
"So far they've looked at the drugs in over 6,000 patients, and there have been no major side effects that have come up," Jampolis says.
Investigators like Cannon are still on the hunt for any potential dangers. "So far, there have been no surprises or any big safety issues," he says.
Some mild side effects in clinical trials include headache, limb pain, and confusion.
Because these drugs are injected, there is also a risk of injection-site reactions, such as redness, swelling, or tiny infections in the skin.
Is this a breakthrough for high cholesterol?
"This is a really exciting new area in cardiovascular medicine, and I think it has a tremendous amount of potential for some patients," Jampolis says.
But she isn't yet convinced that the drugs will completely change the way doctors treat high cholesterol. Statins likely will remain the first choice to treat high cholesterol, she says.
Still, the new drugs have "some really exciting potential implications as far as decreasing risk in people who can't tolerate statins or just don't get the results they need to decrease the risk of a heart attack," she says.
Is there such a thing as too-low cholesterol?
This is the question that seems to give researchers the most pause.
"We haven't seen that yet, but it remains a little bit of a question," Cannon says. "These drugs are so powerful you can get the cholesterol down to zero, and that seems like it's probably not a good idea, but we don't know yet."
"We haven't really been able to get cholesterol down this low before," Jampolis says. "These drugs are very powerful, so these studies that are ongoing are going to be looking very closely at potential side effects from getting cholesterol too low."
Though cholesterol is generally looked at as a bad thing, there are some functions in the body that use this substance for good, she says. For example, cholesterol molecules help convert sunlight on the skin into vitamin D, and it is a vital building block of our cells.
Too much cholesterol, though, can lead to plaque buildup in the arteries and heart disease.
What will they cost?
We don't know yet, but the national pharmacy chain CVS Health estimates that these drugs may cost between $7,000 and $12,000 for a year's supply.
It's unclear which if any insurance companies will cover these meds when they become available.
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