Expert Panel Recommends FDA Approve New Cholesterol Drug

On Tuesday, an FDA advisory panel voted 13-3 to recommend that the agency approve Praluent, an injectable cholesterol-lowering drug, though the panel also said more data are needed to determine the drug's ability to reduce strokes and heart attacks, AP/Modern Healthcare reports (AP/Modern Healthcare, 6/9).

Praluent Details

Sanofi and Regeneron Pharmaceuticals are the manufacturers of Praluent, known generically as alirocumab. The treatment is part of a new class of medicines, often called PCSK9 inhibitors, thought to have the potential to change the course of cardiovascular treatment and result in billions of dollars in revenue for drug companies. Such drugs block the PCSK9 protein, which affects the body's ability to clear "bad cholesterol." Drugs in the class could be beneficial for individuals who cannot take or do not benefit from treatment with statins.

If FDA approves Praluent, it would be the first treatment in the drug class to gain approval, according to the Wall Street Journal. The FDA panel is considering another PCSK9 inhibitor, Repatha by Amgen, on Wednesday.

Most studies have shown that Praluent lowered patients' cholesterol by about 40% to 60%. Patients who took Praluent showed a slightly greater incidence of memory impairment than those in comparison groups. However, the side effect "was not characterized as serious" among the eight patients who experienced such a reaction, according to James Smith, deputy director of the FDA's division of metabolism and endocrinology products. In addition, 2.5% of Praluent patients experienced liver problems, compared with 1.8% of patients taking a placebo (Burton, Wall Street Journal, 6/9).

Panel Discussion

The panel considered whether there was sufficient evidence to recommend the drug be approved before it underwent testing in large clinical trials (Kolata, New York Times, 6/9). Several panelists voiced concern about whether completed trials were large enough to draw conclusions about safety (Firth, MedPage Today, 6/9).

In addition, several panelists said that the drug should be used only by patients with abnormally high levels of cholesterol caused by a certain inherited disorder, according to AP/Modern Healthcare.

Philip Sanger of Stanford University, who voted to approve the drug, said, "I personally fall on the side of having optimism, but I need to see the cardiovascular outcome study to know."

Michele Orzsa, the panel's consumer representative, said, "I am really only comfortable with the risk-benefit equation in that population" (AP/Modern Healthcare, 6/9).

Cost Concerns

Some critics have raised concerns over the cost of PCSK9 inhibitors, USA Today reports. The drugs could cost $10,000 per year (Szabo/Winter, USA Today, 6/9).

The costs, combined with the potential number of patients, could be a source of concern for insurers, as well as others who would have to pay for the drugs, according to the Times. If FDA grants broad approval for Praluent, about 11 million U.S. residents could be eligible to take the drug, according to Sanofi estimates (New York Times, 6/9).