By Thomas M. Burton
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 and Regeneron Pharmaceuticals Inc. 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.
The new injectable medicine is generically called alirocumab and
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.
This new class of medicines are 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 the link is fairly
clear with statin drugs, they worry that this may not be so clear
with the new PCSK9 drugs.
"I don't believe we have enough data today," said Dr. 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, but panel members expressed concern that
this connection may not be so clear with the new class of
medicines.
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 indications to certain patients with genetic high
cholesterol. "I would not allow broader use," he said.
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 it generally does so.
Write to Thomas M. Burton at tom.burton@wsj.com
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