By Jonathan D. Rockoff
A Food and Drug Administration advisory panel this week
considers experimental cholesterol-lowering drugs whose approval
could pave the way for blockbuster medicines with potentially
billions of dollars in sales.
The panel is evaluating evolocumab from Amgen Inc. and
alirocumab from Sanofi SA and partner Regeneron Pharmaceuticals
Inc., new LDL-lowering agents that would be the first major
additions to the coronary heart-disease medicine chest since statin
pills were first prescribed in the late 1980s.
"This is the next big quantum leap in being able to control
cholesterol and potentially reduce the risk of" heart attacks and
strokes, said Bill Sasiela, who heads the cardiovascular and
metabolic program at Regeneron.
Statins, such as Lipitor from Pfizer Inc. and Crestor from
AstraZeneca PLC, will remain a backbone of efforts to lower "bad"
LDL cholesterol and reduce the risk of heart attacks and strokes.
Yet doctors have been looking for another option because millions
of people with high cholesterol can't tolerate statins or get their
condition under control using the drugs.
The new biotech drugs blocks a protein called PCSK9, which
interferes with the liver's ability to clear bad cholesterol. Both
are injections that drug companies say patients will be able to
give to themselves at home. Studies indicate their use dramatically
lowers bad cholesterol levels in patients, and the companies say
studies have shown the drugs to be safe.
One concern, which the FDA has discussed with companies, is
whether use of the drugs could raise the risk for side effects such
as memory impairment or delirium. The companies say they continue
to study the matter, but that so far, testing doesn't suggest a
heightened and significant risk.
Also unclear is whether the cholesterol reductions seen in
patients taking the new agents also reduces their risk of heart
attacks and strokes, though the FDA typically accepts that sizable
reductions in bad cholesterol translate into cutting the
cardiovascular risks. Company-sponsored "outcomes" studies probing
the issue are under way and expected to finish in 2017 or
earlier.
Given the potential size of the market, the PCSK9 class could be
among the industry's biggest-selling new classes. Sales could reach
as much as $10 billion a year world-wide, estimate analysts at
brokerage firm Goldman Sachs Group Inc.
But the drug companies could face a fight from health plans and
drug-benefit managers. The price of the drugs could approach $1,000
a month, according to analysts at Sanford C. Bernstein & Co.
That could means billions of dollars in additional
drug-reimbursement costs for payers, who have already begun making
noises about taking steps to limit use.
"Managed pharmacy care, indeed the health care system, has never
seen a challenge like this to our resilience in absorbing costs,"
CVS Health Corp. executives wrote last February on the blog of the
journal Health Affairs. "Action will take the form of compliance
with clinical guidelines, and careful managed care oversight."
The drug companies wouldn't discuss their pricing plans, but
touted the public-health benefits of preventing heart attacks and
strokes. Helping patients achieve healthy cholesterol levels "will
provide significant medical value," said Jay Edelberg, who heads
PCSK9 development at Sanofi.
"If approved, Repatha would provide patients and physicians with
an important new treatment option for managing high cholesterol,"
Sean E. Harper, Amgen's research and development chief, said in a
statement.
Drug companies have been angling for any competitive advantage
with the new drugs. Sanofi and Regeneron got first dibs on FDA
approval by paying $67.5 million for a voucher that moved up their
decision date to late July, about a month ahead of the deadline for
Amgen's drug. The FDA isn't bound by the recommendations of its
advisory committees but usually follows them.
Last October, Amgen filed a patent suit against Sanofi and
Regeneron asking a federal court to prevent sales of their drug.
Sanofi and Regeneron say Amgen's lawsuit lacks merit.
The companies also have been trying to make the case that the
dosing regimen for their drug is superior to their rivals'. The
companies have been using the brand names for their drugs, which
they often don't do until FDA approval. Amgen's drug will be called
Repatha, while Sanofi and Regeneron call theirs Praluent.
The prospects for PCSK9 sales are bright enough that Pfizer has
been racing to catch up and be part of the conversation about the
drugs. "Given the size and diversity of patients, we think there is
an opportunity to be competitive," said Geno Germano, who heads the
Pfizer business that is developing a PCSK9 injection, pill and
vaccine.
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