ZURICH-- Novartis AG said Monday the U.S. Food and Drug
Administration designated a new leukemia treatment it is developing
as a "breakthrough" therapy, giving it access to a potentially
faster regulatory review process.
Basel-based Novartis's CTL019 treatment, which is being tested
at the University of Pennsylvania's Perelman School of Medicine, is
designed to treat relapsed or refractory acute lymphoblastic
leukemia, a rapidly worsening form of cancer that causes abnormal
white blood cells.
CTL019 is part of a new wave of experimental treatments Novartis
is developing, called CAR therapies. They are a new type of
immunotherapy, an approach which uses the body's own immune system
to fight disease, and which has become the hottest new area of
cancer treatment.
Immunotherapies currently in development by drug companies
including Merck & Co, Bristol-Myers Squibb and Roche Holding
are administered via intravenous drip, but CAR therapies involve
extracting, genetically engineering, and re-injecting the patient's
own blood cells to fight the cancer.
Patients treated with CTL019 have their own T cells--a type of
white blood cell that fights infection--extracted, reprogrammed to
target the disease, and re-injected. Each treatment would be unique
to just one specific individual and the genetic makeup of their
cells.
"Novartis welcomes increased dialogue with the FDA and a
potentially expedited review to streamline the development of
CTL019," David Epstein, who runs Novartis's pharmaceutical
division, said in a statement.
Breakthrough status is given by the FDA to experimental
therapies that demonstrate a substantial improvement over existing
treatments in clinical testing. It can lead to faster regulatory
review, although regulatory approval is by no means guaranteed.
Early-stage clinical trials of CTL019 showed promising results.
Nineteen out of 22 children with acute lymphoblastic leukemia
treated experienced complete remission of the disease, although
five later relapsed. In adults, the remission rate was 7 out of 32,
with 15 responding to treatment.
Write to Andrew Morse at andrew.morse@wsj.com and Hester
Plumridge at Hester.Plumridge@wsj.com
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