FDA Panel

A Food and Drug Administration (FDA) committee today recommended approval of a new biologic drug to lower LDL, so-called “bad” cholesterol.

Officials at the FDA generally follow the recommendations of their advisory committees.

The panel gave the thumbs-up to Sanofi’s drug, Praluent. The committee will analyze a similar drug, Amgen’s Repatha, on Wednesday. Pfizer is developing a drug in the same class, called PCSK9 inhibitors.

PCSK9 inhibitors attack an enzyme, called PCSK9, that slows the body’s ability to clear cholesterol. Like most biologic drugs, they must be injected.

The full FDA will determine whether to limit the drugs to high-risk patients or to approve them for all of the 2 million Americans with high cholesterol that doesn’t respond to dietary changes or statin drugs.

Learn More About PCSK9 Inhibitors »

Cholesterol Is a Killer

LDL cholesterol is a major risk factor for heart attack and stroke, the number one and five killers, respectively, in the United States. The lower the number the better, doctors say. A maximum safe lab result is considered to be 100 mg/dL.

In patients with starting LDL counts well above 100 mg/dL, doctors can rarely bring numbers into the safe range using statins alone. Statins reduce LDL cholesterol by 30 to 50 percent. The new drugs lower it by about half. The two types of drugs could potentially be taken together to spur dramatic reductions in cholesterol.

Cholesterol Drug

With PCSK9 inhibitors, doctors could potentially bring a patient’s cholesterol down to numbers normally seen in children under a year of age.

“If we could change our population mean LDL cholesterol to ranges that we haven’t been able to achieve with any previous form of treatment, maybe it would change the number one killer in the United States,” said Dr. Elliott Antman, president of the American Heart Association.

Will the Drugs Lower the Number of Heart Attacks and Strokes?

There are questions about the new drugs, however.

Makers of the PCSK9 inhibitors have not yet shown that the drugs reduce the risk of heart attack. Researchers had to abort a previous effort to make a biologic cholesterol drug when studies showed that patients who took it in combination with statins had higher death rates.

Statins, by contrast, have been shown to reduce cardiovascular events by 25 to 50 percent over five years.

Antman sees this gap in the evidence as a non-issue.

“We have decades worth of data showing the relation between LDL cholesterol and cardiovascular events in thousands of people,” he said. “You can draw a mathematical relation between how much you reduce your cholesterol and how much you reduce risk of cardiovascular disease.”

Past Coverage: Promising Early Results for Cholesterol-Busting Drugs »

Sticker Shock

The most controversial issue about the drugs is their price tag. Cost estimates for the PCSK9 drugs range from $7,000 to $12,000 per patient annually, according to the pharmacy benefits company Prime Therapeutics.

Statins, many of which are now available in generic form, cost about $250 a year.

That sticker price could add between $0.93 and $6.71 per member per month to insurers’ costs and up to $15.66 per member per month to Medicare coverage costs. The range of costs depends on how narrow a patient population the drugs are approved to treat.

But, said Antman, if the drugs dramatically reduce the number of heart attacks and strokes, they could pay for themselves by reducing the costs of caring for heart attack and stroke patients.

“The unit cost of the individual treatments may be quite high,” he said, but “if you analyze the benefits to the healthcare system by seeing a reduction in the cost of caring for individuals who have heart attacks and strokes, and the economic burden that that represents to our society, this becomes a very interesting calculus.” 

More than 2,000 people die from a heart attack or stroke every day. Survivors often require long-term care, medications and, occasionally, heart transplants.

Why Do Statins Work Better for Some People Than Others? »