* Panel votes 9-3 to ask for more data on Clolar
* Company says current study backs approval for adult use
* Genzyme shares close down 0.6 percent
(Adds company statement in paragraphs 7-8; updates share
By Lisa Richwine
SILVER SPRING, Md., Sept 1 Genzyme Corp
GENZ.O should run a new study before the company can win
approval to promote a pediatric leukemia drug for older adults
with an aggressive blood cancer, a U.S. advisory panel said on
In a 9-3 vote, the committee urged regulators to require a
new study of adults randomly assigned to the Genzyme drug,
Clolar, or another treatment for comparison. Genzyme has
applied for approval of adult use based on a study that did not
include a comparison group.
Several panelists said the drug appeared to have
cancer-fighting ability but Genzyme's study was not sufficient
to establish its level of safety and effectiveness and which
types of patients would benefit.
"In the absence of a randomized trial, we don't have an
evidence-based justification that alternative standard therapy
wouldn't be better," said panel member Thomas Fleming, a
statistician at the University of Washington in Seattle.
Genzyme shares fell 0.6 percent to close Tuesday at $55.39
The panel recommendation makes it unlikely the FDA will
approve Genzyme's current application for use in previously
untreated adults aged 60 and older with acute myeloid leukemia
(AML). The agency usually follows advice from its panels of
outside experts, and FDA reviewers said they had urged Genzyme
in 2007 to run a trial with a comparison group.
Mark Enyedy, president of Genzyme Oncology and Multiple
Sclerosis, said in a statement the company remained committed
to the clinical development of Clolar in older patients, a
population with high unmet medical need.
The company has a study underway comparing Clolar to a
chemotherapy drug called cytarabine in a slightly different
older patient group -- adults aged 55 or older with AML that is
not responding to other treatments. Results in that trial are
expected in 2011.
Clolar is approved for fighting a different type of
leukemia in children, and doctors already use it to treat
adults with AML. Genzyme needs FDA clearance, however, to
market the drug specifically for the adults. The drug's generic
name is clofarabine.
JP Morgan analyst Geoffrey Meacham said the panel vote was
"an incremental negative as it appears unlikely Clolar will be
a meaningful revenue contributor in the near-term."
Meacham, in a research note, said he was not adjusting his
estimates for Clolar sales. He forecast the drug would make up
less than 20 percent of the $550 million expected from
Genzyme's hematologic oncology sales in 2010.
Company officials told the panel they had consulted with
AML experts and could not find an appropriate comparison group
for their clinical trial. They said current data supported the
expanded use of Clolar as 46 percent of 112 patients had their
disease go into remission for the median time of one year.
"These data are compelling," Dr. Michael Vasconcelles, a
Genzyme vice president, told the panel, adding that potential
side effects such as bone marrow suppression and nausea and
vomiting were acceptable.
After the panel meeting, Genzyme Vice President Mark Hayes
said the company had "further opportunity to engage the FDA" in
discussions about the application as the agency had pushed back
its deadline to rule on Clolar in adult AML to December.
(Reporting by Lisa Richwine; Editing by Andre Grenon, Richard
Chang and Tim Dobbyn)