Wed, Jun-10-15, 05:27
To Good Health!
Plan: IF Fung/LC Westman/Primal
All over the evening news of its approval yesterday, in restricted cases. That's really jumping the queue! Many concerns expressed, but not to worry, just a touch of memory impairment. :-(
By THOMAS M. BURTON
Updated June 9, 2015 7:56 p.m. ET
A Food and Drug Administration advisory panel recommended that the agency approve the cholesterol-lowering drug Praluent, the first of a wave of such cardiovascular drugs expected to raise billions of dollars in revenue and perhaps alter the treatment of cardiovascular disease.
But many panelists said the use of the drug should be limited to certain high-risk groups, such as people with very high cholesterol for genetic reasons because of a condition called familial hypercholesterolemia.
The committee voted 13-3 in favor of Praluent, from France’s Sanofi SA and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. But enough panelists expressed caution about the evidence in the companies’ studies, and so it may take longer than the industry would like to get these medicines widely used.
Panel member Brendan M. Everett, director of the cardiology inpatient service at Brigham and Women’s Hospital in Boston, said he would limit the drug’s immediate use to certain patients with genetic high cholesterol. “I would not allow broader use,” Dr. Everett
The new injectable medicine is administered every two weeks, and is generically called alirocumab. It could help some patients who can’t tolerate or aren’t effectively treated with statin drugs such as Lipitor. Another such drug, Repatha from Amgen Inc., will go before the panel on Wednesday.
The panel’s recommendation late Tuesday boosted shares of Sanofi, which was up 3% in after-hours trading at $50.92. Shares of Regeneron had been halted in regular trading Tuesday pending news.
This new class of medicines is often called PCSK9-inhibitors, because they block a protein called PCSK9, which interferes with the liver’s ability to clear so-called bad cholesterol from the bloodstream. That cholesterol, called LDL, is linked to cardiac disease, albeit imperfectly.
Several members of the panel said that while statin drugs have been shown to lower cholesterol and reduce cardiac disease, it’s not clear if these new PCSK9 drugs will do the same. “I don’t believe we have enough data today,” said William R. Hiatt, a cardiologist at the University of Colorado School of Medicine. Statin drugs have been used by millions of people for more than two decades.
The committee’s chairman, Robert J. Smith of Brown University, also expressed the wider concern among several panel members that the effect of LDL “may be different for different patient groups.”
The two companies say they already have a study under way to see if cardiac outcomes improve with the drug, and results are expected in 2017.
The link between LDL and cardiac events like heart attack and stroke isn’t exact, although it has been shown to be significant in a number of cardiovascular studies previously.
One impediment to use of the drug, if it should win FDA approval, is its likely cost. Some Wall Street analysts say such drugs could cost about $1,000 monthly.
The FDA doesn’t always follow the recommendations of its advisory committees, but generally does so.
In clinical studies evaluated by the FDA and advisory committee, Praluent showed “no marked disparities in deaths, serious adverse events or adverse events leading to discontinuation” of the drug, wrote James P. Smith, deputy director of the FDA’s division of metabolism and endocrinology products in the agency’s drug center.
He wrote that there was some memory impairment in a greater incidence of patients with Praluent than in comparison-group patients, but that “was not characterized as serious” in the eight patients reporting this reaction. Also, there were more liver problems with Praluent, with 2.5% of patients suffering them, compared with 1.8% on placebo.
Regarding the effectiveness of the drug, most of the studies showed patients with the drug had their cholesterol lowered by approximately 40% to 60%.
Reviewers for the FDA, in a report for the panel, said the drug “demonstrates early and sustained” lowering of LDL cholesterol, “regardless of background lipid-modifying therapies and is generally well-tolerated.”
Various members of the expert panel, while saying they were overall comfortable with the data on the drug, expressed the concern that studies so far hadn’t gone on for long enough. Kenneth D. Burman, a professor of medicine at Georgetown University and a panel member, said “it seems probable that the drug will be safe over time,” but that, “We need longer-term studies.”
Write to Thomas M. Burton at tom.burton~wsj.com