(In Nov 13 story "UPDATE 2- FDA panel supports approval of
Sanofi's MS drug Lemtrada," corrects headline, paragraphs 1-2 to
clarify that panel said safety concerns should not preclude
approval despite risk of side effects, from panel "recommended
approval"; corrects paragraph 2 to show vote was 17 to 0, not
14-0, removes reference to surprise decision; clarifies in graph
1, adds new graph 3, to show panel concerns about studies;
removes graph 1 reference to reserving drug for "patients who
have failed other therapies"; adds Maryland to dateline.)
By Deborah Zabarenko
SILVER SPRING, Maryland Nov 13 A federal
advisory panel said safety issues do not preclude approval of
Sanofi SA's experimental multiple sclerosis drug
Lemtrada on Wednesday, but voiced concerns about the quality of
An advisory panel to the Food and Drug Administration voted
17 to 0, with one abstention, on Wednesday that the drug's
potential to cause cancer and other serious conditions was not a
reason to prevent patients from gaining access to it.
However, a majority of panel members believed Sanofi's
studies were not adequate and well-controlled, and they said
that while it appeared to be effective in patients with
relapsing MS it had not been proven to help against disability.
"Do I want to take this drug? No way!" said Dr. Nathan
Fountain of the University of Virginia School of Medicine and
the panel's chairperson. But for some patients, he said, it
could be appropriate. "I wouldn't want to deny those people," he
The panel's recommendation follows an initial report last
week by reviewers for the FDA, who raised grave concerns about
the drug's potential to cause an array of autoimmune conditions,
in which the body mistakenly attacks its own cells, as well as
its potential to cause thyroid, skin and breast cancer.
Advisory panelists, who met in Silver Spring, Maryland, said
the risks could be worth it for some difficult-to-treat
"The risks are very substantial, but this is a really bad
disease," said Dr. Paul Rosenberg of the Johns Hopkins
University School of Medicine.
Multiple sclerosis is a chronic, autoimmune disease that
affects more than 2.5 million people worldwide and up to 500,000
in the United States, according to the Multiple Sclerosis
Foundation. It can cause muscle weakness, pain, and speech and
Lemtrada is designed to treat relapsing remitting multiple
sclerosis, the most common form of the disease, in which
flare-ups are followed by periods of recovery.
The drug, which was approved in Europe in September, was at
the heart of Sanofi's lengthy, $20.1 billion takeover battle for
Genzyme Corp., which developed the drug. Sanofi finally acquired
Genzyme in 2011.
As part of the takeover deal, Genzyme shareholders received
contingent value rights, known as CVRs, entitling them to future
payments of up to $14 a share if certain goals were met,
including approval for Lemtrada.
The price of the CVRs has fallen 67 percent to 66
cents since the FDA posted its review last week.
If Lemtrada, also known as alemtuzumab, is approved, it is
expected to generate peak sales of $752 million by 2018,
according to seven analysts polled by Thomson Reuters.
The FDA is not bound to follow the advice of its advisory
committees but typically does so.
Two late-stage clinical trials showed that patients taking
Lemtrada were significantly less likely to experience a relapse
over the course of two years than patients taking Rebif, a
standard therapy made by EMD Serono, a subsidiary of Merck KGaA
and Pfizer Inc.
But patients and investigators knew which treatment they
were getting, raising questions as to whether the results could
have been biased. Sanofi argued that it was difficult to
disguise who was taking which treatment given the differences in
how they are dosed.
Lemtrada is a monoclonal antibody that is thought to
selectively deplete T and B cells, while sparing other
infection-fighting elements of the immune system. It is given
intravenously for five days and then for three days a year
If the drug wins approval it would likely compete most
closely with Biogen Idec Inc's Tysabri, which is one of
the most effective drugs for multiple sclerosis on the market
but can cause a potentially fatal brain infection known as PML.
That risk has limited Tysabri's usage to patients who have
failed other treatments -- a group which Lemtrada, if approved,
would also target.
Most MS drugs are either injected or infused. The market is
increasingly moving toward oral treatments, of which Biogen's
recently launched Tecfidera is expected to be the market leader,
with peak annual sales of more than $3 billion.
Oral drugs are expected to eventually take market share from
injectibles, which also include Teva Pharmaceutical's Copaxone.
(Reporting by Deborah Zabarenko in Silver Spring, Maryland;
writing by Toni Clarke; Editing by Leslie Adler)