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FDA Approves Pricey New Cholesterol Drug

Community Outreach Group Provides Health Screening To NYC Bodega Owners

A cholesterol check during a free health-screening clinic for New York bodega convenience store workers. The FDA just approved the first in a new class of cholesterol drugs. Getty Images file

The Food and Drug Administration has approved the first of a new class of cholesterol drugs aimed at helping people with a hard-to-treat, inherited form of high cholesterol.

The drug, called Praluent, must be injected, so it won't be as easy to take as a pill. Because it's in a class of biotech drugs called monoclonal antibodies, it will be pricey — $14,000 a year.

It's in a new class of drugs known as proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors. It targets PCSK9, a protein that affects the liver's ability to take LDL or "bad" cholesterol out of the blood.

"Praluent is approved for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia or patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol," the FDA said in a statement.

"Multiple clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke," the FDA added. A trial is going now to see if adding Praluent to statins affects the risk.

"The most common side effects of Praluent include itching, swelling, pain, or bruising where injection is given, nasopharyngitis, and flu," FDA said.

It's given as an injection once every two weeks.

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The drug is likely to be prescribed to people who cannot tolerate statins, as well. Once a drug is approved, doctors may prescribe it for anything they like.

Richard Arnstine, a 60-year-old orthodontist living in Cleveland, has a bad family history of heart disease and he's one of about 10 percent of people who get a painful, muscle-damaging side-effect from statins.

"I got to the point where at times I literally couldn't open a door because my muscles were so sore," Arnstine told NBC News.

He couldn't sleep at night because his legs ached so much, and found he couldn't work because it was painful to stand and to use his hands.

"I really just had to quit and I would tell my cardiologist at that time that it was either stopping practicing my chosen profession, or taking the statins. I chose to continue to practice," he said.

He's tried diet and exercise but still has evidence that his arteries are clogging up.

"I'm about to be a grandfather in about another eight weeks so I surely want to be around for my grandchildren and for my wife and my other children," he said.

Some groups, including large health insurers, have complained that the PCSK9 inhibitors will be expensive. Statins are available generically and can be very inexpensive.

But the drug maker's lobbying group, PhRMA, says the new drugs are only for a limited group of patients.

"Despite claims that tens of millions of Americans could be eligible for treatment with PCSK9 inhibitors, these medicines will not replace existing treatment options and address a significant unmet medical need in a small subset of people," PhRMA said in a statement.

But research firm GlobalData says the drugs have "extraordinary blockbuster potential" and could generate $17.5 billion in sales by 2023, in part because they will be so expensive.

Praluent is made by Sanofi and Regeneron. An FDA committee also recommended approval of a second drug in the same class, made by Amgen. European regulators have approved that one.

"Actual costs to patients, payers and health systems are anticipated to be lower as ... pricing does not reflect discounts or rebates," Regeneron said in a statement.

"Out-of-pocket costs to patients will vary depending on insurance status and eligibility for patient assistance."

Just this week, a group of more than 100 oncologists made a public plea for cheaper cancer drugs. They urged patients to lobby Congress and drug companies and said the federal insurance program Medicare should get the power to negotiate for lower drug prices.

"Despite statins and other lipid-lowering therapies, many patients are unable to reach their LDL cholesterol goals, and may benefit from new therapeutic options such as Praluent," said Dr.Elias Zerhouni, president of research and development for Sanofi and a former director of the National Institutes of Health.

Heart disease is the No. 1 killer in the U.S. and many other countries. It's caused in large part by unhealthy cholesterol levels, as well as by high blood pressure and smoking.

NBC's Erika Edwards contributed to this story.