Novartis Wins 'Breakthrough' Status for Leukemia Treatment

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 [e-mail] and Hester Plumridge at [e-mail]

Subscribe to WSJ: http://online.wsj.com?mod=djnwires