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FDA Panel Unanimously Endorses Novartis AG (NVS)'s CAR-T Leukemia Drug



7/13/2017 5:40:19 AM

FDA Panel Unanimously Endorses Novartis' CAR-T Leukemia Drug July 13, 2017
By Mark Terry, BioSpace.com Breaking News Staff

A U.S. Food and Drug Administration (FDA) advisory panel unanimously recommended approval of Novartis (NVS)’ CAR-T therapeutic, CTL019 (tisagenlecleucel) for B-cell acute lymphoblastic leukemia.


If approved, it will be the first gene therapy approved in the U.S. Although the FDA isn’t required to follow the recommendation of the review panels, it usually does. Approval is likely in September. The panel unanimously voted 10 to 0 to recommend tisagenlecleucel for approval.

The FDA’s Oncologic Drugs Advisory Committee (ODAC) evaluated Novartis’ chimeric antigen receptor T cell (CAR-T) therapeutic to treat relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). The committee didn’t spend much time evaluating the drug’s effectiveness, which has been dramatic, but instead focused on possibly life-threatening side effects, which include cytokine release syndrome and neurological toxicity that can result in seizures and potentially death.

James Gulley, from the National Cancer Institute (NCI), one of the panel members, noted, “This is a novel therapy, there’s an unmet medical need, a strong efficacy and a good risk mitigation strategy.”

Another panel member, Timothy Cripe, an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called it “the most exciting thing I’ve seen in my lifetime.”

CAR-T is a laborious process unique to each patient. T-cells, a type of immune cell, are collected from the patient’s blood. They are then frozen and shipped to a Novartis facility in Morris Plains, N.J. There, the cells are genetically engineered to attack the specific cancer, multiplied, refrozen, and shipped back to the patient’s physician to be infused into the patient. Once they are infused, the T-cells quickly multiply and are already super-stimulated to attack the patient’s specific leukemia and lymphoma cells. The turnaround time, which is dubbed “vein-to-vein,” is estimated at about 22 days.

The Washington Post notes, “Most patients in the Novartis study experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to extremely severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in the study also had neurological problems, including seizures and delirium. But there were no cases of fatal brain swelling, as occurred in another company’s trial, Grupp said.
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That company is Juno Therapeutics (JUNO), which was once leading the race to develop a successful CAR-T therapeutic.

Novartis has developed a set of protocols to deal with the potential side effects, which include limiting it to 30 to 35 medical centers in the U.S. where oncology departments receive extensive training with the therapy. It also will have Novartis staffers at those centers and plans to follow each patient for up to 15 years.

Because of the labor-intensity of CAR-T therapy, the price is likely to be high. Some projections for CAR-T range around $300,000 for a treatment, although Novartis hasn’t released any pricing information yet.

In this case, payers are unlikely to blink at the high price. The trial studied 63 patients, and 82.5 percent, 52 of them, went into remission. Eleven patients, however, died. But the cancer it is being approved for is often fatal in children, and current treatments are also expensive, run on for several years, and involve repeated hospitals stays that can far exceed the prices currently being projected.

“The panel’s unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need,” said Bruno Strigini, chief executive officer of Novartis Oncology (NVS), in a statement. “We’re very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review.”

In addition to the press and numerous oncologists, the panel audience included patients and their families who had successfully received the treatment. One was Emily Whitehead and her father Thomas. Emily was the first pediatric patient to receive the treatment when she was six years old with leukemia. At that time, The Washington Post notes, she was close to death. “The treatment almost killed her, but she recovered and today is cancer free.”

“If you want to see what a cure looks like for relapsed ALL, she’s standing right beside me,” Thomas Whitehead said.


Read at BioSpace.com


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