New drug could dramatically reduce cholesterol

The Food and Drug Administration on Thursday approved the second in a new class of cholesterol drugs that promises to dramatically lower cholesterol, although at a very high price.

Amgen's Repatha, also known generically as evolocumab, belongs to a new class of injectable cholesterol fighters called PCSK9 inhibitors, which are man-made antibodies. The FDA last month approved the first of these drugs, Praluent, made by Sanofi and Regeneron Pharmaceuticals.

Both drugs will cost more than $14,000 a year.

That's far more than statins, the dominant cholesterol drug, which can cost only $250 for generics. The costs of the new cholesterol fighters is compounded by the fact that patients would be expected to take them for the rest of their lives.

The FDA granted both Repatha and Praluent broad approval, allowing it to be prescribed along with statins to people with cardiovascular disease, such as those who have had heart attacks or strokes, who need to lower their LDL.

In a clinical trial, patients who combined Repatha with statins lowered their cholesterol by 60%, compared to placebo. Side effects include sore throats, upper respiratory tract infections, flu, back pain and bruises or sore where the injections are given, according to the FDA.

"Cardiovascular disease is a serious threat to the health of Americans, and the FDA is committed to facilitating the development and approval of effective and safe drugs to address this important public health problem," said John Jenkins, director of the Office of New Drugs at the FDA's Center for Drug Evaluation and Research.

But there's no evidence yet that Repatha actually prevents heart attacks, strokes or death, said cardiologist Cam Patterson, chief operating officer at New York-Presbyterian Hospital/Weill Cornell Medical Center. Doctors also don't yet know what side effects Repatha will cause when it's taken by millions of Americans, not just the small number of patients in a clinical trial.

"The FDA is really setting a new norm" by approving cholesterol drugs that have no evidence of actually preventing heart attack, Patterson said.

Doctors won't really know the best drugs to prescribe for their patients, Patterson said.

Up to 11 million Americans could be eligible for the new class of cholesterol medications, according to Amgen.

This class of drugs "have the potential to grow over the next several years to become the costliest therapy class our country has seen," said Steve Miller, senior vice president and chief medical officer at Express Scripts, a leading pharmacy benefit manager, in a statement.

The USA already spends $300 billion on medications, Miller said in an interview. Those costs can hit taxpayers, not just consumers, as Medicare, Medicaid and other public health programs struggle to keep pay for new medications.

Having two PCSK9 inhibitors on the market will allow companies to pit them against each other to bargain for the best deal, Miller said. That should save consumers a lot of money.

Express Scripts was able to bargain with the makers of new drugs for hepatitis C, for example, saving its clients more than $1 billion in 2015 and setting off a pricing war between drug makers, Miller said.

With two drugs on the market, "we've got to hope this gives the consumer some power," Patterson said. "But historically, whenever new drugs come into the market, consumers are never the ones to be in the driver's seat."

Even with steep discounts, the new cholesterol drugs could still cost thousands of dollars a year, Miller said. That means that insurance plans are likely to put strict limits on which patients are eligible for them.

If the drugs really do prevent heart attacks and strokes, and if they're used only by patients who really need them – rather than anyone with high cholesterol -- they could save money by keeping patients out of the hospital, Miller said.