New cholesterol drug OKd

WASHINGTON - The Food and Drug Administration on Friday approved a first-of-a-kind drug that lowers artery-clogging cholesterol more than older medications that have been prescribed for decades.

The drug offers an important new option for millions of patients at high risk of heart disease. But its sky-high price tag - $40 a day - is certain to escalate debate about the cost of breakthrough drugs and who should take them.

Praluent is the first in a new class of biotech medications, known as PCSK9 inhibitors, that use a novel approach to lower bad, or LDL, cholesterol. They are considered the first major advance in treatment since the introduction of statin drugs more than 20 years ago, and analysts expect them to generate billions in sales.

But the prospect of introducing highly expensive, injectable drugs for one of the most common medical conditions is drawing concerns from health insurers, doctors and employers, especially since generic statin pills are available for as little as a dime a day.

More than 73 million U.S. adults, or nearly one-third, have high LDL cholesterol, according to the Centers for Disease Control and Prevention. Those patients have twice the risk of heart disease, the leading cause of death worldwide.

Praluent was developed through a partnership between Sanofi, with U.S. headquarters in Bridgewater, N.J., and Tarrytown, N.Y.-based Regeneron.

The FDA approved it for two groups of people at highest risk of heart problems:

Patients with abnormally high LDL cholesterol caused by an inherited condition.

Patients with high LDL cholesterol and a history of heart attack, stroke, chest pain and related conditions.

Sanofi estimates those groups account for 8 million to 10 million patients in the United States.

The price for the new drug that Sanofi announced on Friday was nearly 50 percent higher than many analyst predictions, which centered on $10,000 a year.

In an interview, Sanofi executive Jay Edelberg said the $14,600 price reflects the "value to the health care system and the value to individual patients." He noted that estimated costs for a patient who has suffered a heart attack or similar cardiovascular problem range from $50,000 to $119,000 over one year.

But experts say more data are needed to establish if Praluent's cholesterol-lowering ability actually translates into fewer heart problems and deaths. Sanofi and Regeneron are conducting an 18,000-patient study to answer that question, but it won't be complete until 2017. Until then, many experts say new drugs like Praluent should be limited to the highest risk patients.

"What we know so far is that they reduce cholesterol really well, but what we're not sure about is whether they actually reduce someone's risk," said Harlan Krumholz, a Yale University cardiologist. "I think the wisest thing is to be cautious about their use and reserve them for people who have no other choice."

An estimated 6.3 million U.S. patients cannot control their LDL levels with statins alone, according to medical surveys. Another 3.3 million are thought to be unable to tolerate statins due to side effects, but there are no formal criteria to identify these patients.

"I think we need to be clear what qualifies for statin intolerance," Neil Stone, a cardiologist at Northwestern University, said. "What moves you from an inexpensive, generic statin to a very expensive new drug that doesn't have near the track record of the statin?"

The FDA is scheduled to rule on a similar drug from Amgen by Aug. 27; it won approval from European regulators last week. Pfizer's entry into the field is expected to launch in 2018 or later.

All three drugs lower low-density lipoprotein, or LDL, cholesterol more powerfully and in a different way than statins. They block a substance called PCSK9, which interferes with the liver's ability to remove cholesterol from the blood. Adding the new drugs to older statins reduces LDL cholesterol by about 40 percent to 60 percent. Statins alone generally lower levels of the wax-like substance by about 25 to 35 percent.

Statistical analyses published earlier this year suggest patients taking PCSK9 drugs have half the risk of dying or suffering a heart problem as patients receiving statins or older drugs. But definitive studies are still ongoing.

For now, several factors could limit use of the drugs. They must be self-injected by patients, with Praluent dosed every two weeks. And their price is expected to face pushback from insurers and other companies that manage drug expenses.

Praluent will cost far less than expensive specialty medications for cystic fibrosis, certain cancers and hepatitis C. But there is potentially a massive collective cost given that the new drug and others in its class could eventually treat large numbers of heart patients and must be taken indefinitely.

CVS Caremark, the giant pharmacy benefit manager, has warned that if 10 million U.S. patients ultimately take PCSK9 drugs it could result in well over $100 billion in new drug spending.

"The one thing you can say for sure is that these medications are going to add substantially to the overall cost of health care," said Troyen Brennan, CVS's chief medical officer.

Analyst predictions for the drugs are more modest, at least initially. Credit Suisse analyst Vamil Divan estimates sales for the entire class - including drugs from Amgen and Pfizer - will reach $10 billion by 2019. Pharmaceutical research firm GlobalData projects global sales of $17.8 billion by 2023.