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 The approval was widely expected after an FDA advisory panel last
month unanimously recommended the action.
Novartis shares closed virtually unchanged in Swiss trading.
Novartis also announced an agreement with the U.S. Centers for
Medicare and Medicaid Services under which payment for the therapy
will be based on clinical outcomes achieved.
The treatment, called Kymriah, was approved for patients up to 25
years of age who have relapsed or not helped by prior treatment for
B-cell acute lymphoblastic leukemia (ALL).
Dr Kevin Curran, a pediatric oncologist at Memorial Sloan Kettering
Cancer Center in New York, noted the treatment's high cost.
"Of course, we have to talk about pricing from a national
standpoint," he said. "But if I have a parent and a (sick) child in
front of me, and I have an opportunity to save them, we're going to
take that."
Kymriah belongs to a new class of treatments called CAR-T therapies.
It involves removing disease-fighting T cells from a patient,
genetically modifying them to better recognize and attack cancer,
and then replacing them, where they can circulate for years seeking
out the disease.
Novartis estimates some 600 ALL patients a year would be eligible
for Kymriah. It expects to open five treatment centers within days
and 35 by year-end.
"We’re entering a new frontier in medical innovation with the
ability to reprogram a patient’s own cells to attack a deadly
cancer,” Food and Drug Administration Commissioner Scott Gottlieb
said in a statement.
The FDA said it has granted 76 applications for trials involving
experimental CAR-T therapies.
Shares of Gilead Sciences Inc, which this week announced an $11.9
billion deal to buy Kite Pharma, were up 6.2 percent to $80.47 on
Wednesday afternoon, as Kite is widely expected to receive the next
U.S. approval of a CAR-T therapy for a different blood cancer.
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Shares of Bluebird Bio Inc, which is developing a promising CAR-T
treatment, were up 11.1 percent at $113.73.
"Two years ago many people would have told you these types of
treatments were science fiction," said Brad Loncar, chief executive
of Loncar Investments, which runs the Loncar Cancer Immunotherapy
ETF
In clinical trials, CAR-T therapies have shown remarkable efficacy
against blood cancers. In the pivotal Novartis trial, 83 percent of
patients achieved remission with a disease that has historically
poor outcomes.
"We've never seen anything like this before and I believe this
therapy may become the new standard of care for this patient
population," Dr Stephan Grupp of Children's Hospital of Philadelphia
said in a statement.
However, this type of therapy carries risk of severe side effects.
Kymriah will have a boxed warning for cytokine release syndrome, a
potentially lethal systemic response to the activation and
proliferation of CAR-T cells, causing high fever and potential for
neurological problems.
Shares of Juno Therapeutics Inc, which last year reported a handful
of patient deaths during trials of its CAR-T therapy, were down 8.9
percent at $39.92.
(Reporting by Bill Berkrot and Mike Erman in New York, Natalie
Grover in Bengaluru, Deena Beasley in Los Angeles and Julie
Steenhuysen in Chicago; Editing by Shounak Dasgupta and Matthew
Lewis)
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