FDA Approves Sanofi/Regeneron Cholesterol Drug with Limits

The U.S. Food and Drug Administration approved a potent new cholesterol-lowering drug from Sanofi SA (SASY.PA) and Regeneron Pharmaceuticals Inc (REGN.O) on Friday but limited its approved use to patients with a hereditary form of the condition and those with cardiovascular disease.

The FDA ruling came just hours after European regulators recommended approval for broader use of the drug, called Praluent, in all patients with high cholesterol who are not able to lower it even by taking maximal doses of statin therapy.

The companies expect the drug to receive formal approval by the European Commission in late September. The commission recently approved a rival drug, Repatha, made by Amgen Inc (AMGN.O).

The FDA approved Praluent for patients with clinical atherosclerotic cardiovascular disease, including those who have had heart attacks or strokes. It also approved the drug for heterozygous familial hypercholesterolemia (HeFH), the most common hereditary form of high cholesterol.

Regeneron said between 8 and 10 million people fall into those categories.

Investors had hoped the FDA would also approve the drug for patients with non-hereditary high cholesterol who cannot tolerate, or do not respond to, statins such as Pfizer Inc's (PFE.N) Lipitor. Statins are widely available in cheaper generic form.

"Investors are citing the lack of inclusion of primary prevention as more narrow than expected," said Mark Schoenebaum, an analyst at Evercore ISI. Shares of Regeneron fell 2.7 percent to $541.85.

Praluent is given every other week by injection in doses of 75 mg or 150 mg. Regeneron said both doses of its drug will be priced wholesale at $1,120 for a 28-day supply. That equates to roughly $14,560 a year.

Some analysts had expected the company to charge less for the lower dose, and had expected a lower price overall, even after rebates are taken into account.

"Assuming 15 percent gross to net this would imply a net price of $12,400 a year, while we think investors were anticipating a net annual price of approximately $10,000 a year," said Phil Nadeau, an analyst at Cowen and Co.

America's Health Insurance Plans, the industry trade group, protested the high price.

"While the FDA's focused guidance recognizes the safety and effectiveness of this treatment for certain patients, the exorbitant price raises concerns as to whether consumers and the health system can sustain the long-term costs," the group's interim chief executive, Dan Durham, said in a statement.

Praluent and Repatha are the first of a new class of LDL-lowering drugs that inhibit a protein known as PCSK9.

On July 21, European regulators approved Repatha with a broad label covering patients with all familial hypercholesteremia, including the rare, homozygous form, and those with non-hereditary high cholesterol.

The FDA is expected to rule on Repatha by Aug. 27. Amgen's shares fell 3.4 percent to $158.59.

Repatha is given as a 140 mg injection every other week or as a monthly injection of 420 mg.

In limiting the approved patient population for Praluent, the FDA followed the advice of its advisory panel, which in June recommended use be limited to patients with a genetic predisposition to high cholesterol and those at high cardiovascular risk who are on maximum doses of statins.

Dr. Kim Allan Williams Sr., president of the American College of Cardiology (ACC), urged physicians to limit use of the drug to high-risk patients until results come in from trials on whether the drug's ability to reduce cholesterol translates into a lower rate of heart attacks and strokes.

"The ACC eagerly awaits the results of clinical trials that are in progress," Williams said in a statement. "In the meantime, we continue to recommend physicians limit prescribing to the very high risk, hard-to-treat group approved by the FDA."

Physicians can prescribe drugs "off label" to any patient even if the FDA has not approved it for that patient group, but insurance companies may not pay and companies cannot market the drugs for unapproved uses.

CVS Health Corp (CVS.N), whose pharmacy benefit division negotiates drug prices for 65 million plan members, has warned it will require patients to obtain approval before it will pay for the drugs.

"We will try to avoid situations in which a patient has not had a good trial of a statin medication or situations where patients appear to be intolerant but there is no biochemical evidence of problems with liver or muscle enzymes," Dr. Troyen Brennan, CVS's chief medical officer, said in an interview

CVS and other large payers such as Express Scripts Holding Co (ESRX.O) put pressure on Gilead Sciences Inc (GILD.O) to lower the price of its $1,000 hepatitis C pill after it was launched in late 2013. Gilead was forced to discount its price after a rival drug from AbbVie Inc (ABBV.N) emerged late last year.

Praluent and Repatha are expected to generate annual sales of more than $2 billion by 2020, according to Thomson Reuters data.

(Reporting by Toni Clarke; Editing by Bernard Orr; and Peter Galloway)

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