By Toni Clarke
Feb 8 U.S. regulators have approved a new drug
from Celgene Corp for patients with multiple myeloma
whose disease has worsened after being treated with other cancer
The U.S. Food and Drug Administration said on Friday it
approved the drug, Pomalyst, also known as pomalidomide, for the
most difficult-to-treat patients.
Celgene, in an emailed statement, said the drug was priced
at about $10,500 per cycle, and clinical trials have shown that
the typical course of treatment is about five cycles.
The company filed its application based on a mid-stage, or
Phase II, trial in which patients either took pomalidomide, plus
a low dose of the standard treatment dexamethasone, or
Of 191 patients evaluated, 34 percent treated with the
two-drug combination responded to treatment, compared with 13
percent of patients treated with pomalidomide alone. The
analysis of whether patients in the treatment group lived longer
has not yet been completed.
Data from the trial, known as MM-002, showed that median
progression-free survival, or the length of time patients lived
before relapsing, was 4.7 months in the pomalidomide plus
low-dose dexamethasone arm, versus 2.7 months in the
The FDA will require the Pomalyst label to carry a "boxed
warning" alerting patients and healthcare professionals that the
drug should not be given to pregnant women because it can cause
life-threatening birth defects, and that it can cause blood
Celgene is also required to institute a risk management
program for Pomalyst, which is designed to work by enlisting the
body's immune system to attack cancer cells.
The new drug comes from the same family as Celgene's
existing drug Revlimid but appears to help patients who have
become resistant to current therapies, including Revlimid and
its predecessor drug Thalomid.
Multiple myeloma is a cancer that starts in plasma cells in
bone marrow and ultimately disrupts the production of normal
blood cells. The American Cancer Society estimates there will be
about 22,300 new cases diagnosed in 2013 and 10,700 deaths.
A variety of new drugs have entered the market during the
last few years, often allowing patients to live years longer.
Last July, for example, the FDA approved Kyprolis, a drug made
by Onyx Pharmaceuticals Inc for patients with multiple
myeloma who had received at least two prior therapies.
"Pomalyst marks the second treatment to receive accelerated
approval in the last seven months, which is unparalleled in any
other cancer," the Multiple Myeloma Research Foundation said in
In addition to MM-002, Celgene conducted a late-stage trial
known as MM-003 in Europe that tested pomalidomide plus a low
dose of dexamethasone versus a high dose of dexamethasone alone.
Results of that trial have been submitted to European regulators
and the company expects to hear whether the drug has been
approved there later this year. They were not included in the
package submitted for FDA approval.
Data from the European trial showed patients who had failed
an average of five previous therapies who took pomalidomide and
low-dose dexamethasone survived significantly longer than those
who took high-dose dexamethasone.
The median overall survival when the data was reported in
November had not yet been reached in the pomalidomide group,
while the median overall survival in the control arm was 34
weeks. The results show that no matter what occurs during the
remainder of the trial, the improvement in overall survival will
be highly statistically significant.
Geoff Meacham, an analyst at J.P. Morgan, has estimated
pomalidomide could generate sales of around $450 million by
2015. Celgene's chief executive has said he expects the drug to
ultimately generate sale of more than $1 billion.