* Panel votes 9-0 that company trial was inadequate
* FDA usually follows panel recommendations
* Shares fall 57 percent
(Adds panel member, company comments, share movement)
By Lisa Richwine
GAITHERSBURG, Md., March 22 Cell Therapeutics
(CTIC.O) suffered a setback on Monday as a U.S. advisory panel
said the company had not collected enough data to win clearance
for an experimental lymphoma drug.
Advisers to the Food and Drug Administration, in a 9-0
vote, said the company's single trial of pixantrone was
inadequate to back marketing approval.
"I don't see this as being a well-designed or well-executed
study," said Dr. Wyndham Wilson, a panel member and a lymphoma
specialist at the National Cancer Institute.
The company's shares fell 57 percent to 39 cents in
afternoon Nasdaq trading. Shares had been halted ahead of the
The panel decision makes it unlikely the FDA will approve
pixantrone without additional data. The agency usually follows
panel recommendations, and FDA scientists who spoke to the
committee also were critical of the company's study.
Cell Therapeutics wants to sell pixantrone under the name
Pixuvri for treating non-Hodgkin's lymphoma that has stopped
responding to other treatments. The blood cancer affects about
66,000 Americans annually.
The company said pixantrone offered an effective therapy
for patients whose cancer worsened after at least two prior
chemotherapy regimens. Patients at that stage have no approved
treatments and often live less than six months.
In the Cell Therapeutics study of 140 patients, 20 percent
had a major decrease in their disease if they got pixantrone,
compared to about 6 percent with a different medicine.
"It is clear to me pixantrone provides an important benefit
in these very poor prognosis patients," said Dr. John Leonard,
a cancer specialist at Weill Cornell Cancer Center and a Cell
But FDA reviewers questioned the company's conclusions.
They said the study tested less than half the number of people
originally planned and included just eight U.S. patients. The
agency also said heart damage and decreased white blood cells
were more common with pixantrone versus other cancer drugs.
Despite their concerns, several panel members said they saw
signs the drug could be helpful and urged additional study.
"This drug has some activity. I don't think anybody on this
committee would debate that point. The population enrolled in
this study, however, doesn't reflect the U.S. population of
lymphoma patients," said Dr. Mikkael Sekeres, an oncologist at
the Cleveland Clinic.
In a statement after the panel vote, Cell Therapeutics
Chief Executive James Bianco said the company was "committed to
working closely with the FDA to address the committee's
comments as quickly as we can."
"We continue to believe that pixantrone should be
considered as a treatment option for patients" with aggressive
NHL, he said.
A final FDA ruling is due by April 23. Swiss drugmaker
Novartis NOVN.VX has an option for a worldwide license to
develop and sell the drug.
(Reporting by Lisa Richwine)