Novartis' CAR-T therapy gets nod from FDA committee

Eleven patients died - four from side effects and seven from the cancer.

Novartis said the entire process will take 22 days by the time it is launched. Though the treatment has posted excellent results among patients where traditional treatment paths have failed, CAR-T has been considered an experimental treatment. Children with cancer are closely monitored for years in clinical practice, said Timothy Cripe, chief of the hematology/oncology division at the Nationwide Children's Hospital. Kite Pharma also has a CAR-T therapy under FDA review and Juno Therapeutics and others are in late stages of testing. But the young patients who would receive it have few alternatives, said Bruce Roth, an oncologist at Washington University School of Medicine in St. Louis, Missouri, and those alternatives carry risks of their own. CTL-019 should give kids with ALL a chance for long-term survival, he said. ODAC members expressed satisfaction with Novartis' plan to minimize risk, which includes limiting distribution of the therapy to selected centers experienced with vehicle T-cell therapy (at least initially) and plans for extensive, long-term postmarketing surveillance. The CAR-T space should start to heat up once other companies start to win FDA approval for their CAR-T therapies.

If approved, tisagenlecluecel would be the first CAR-T therapy cleared for commercial use anywhere in the world, putting Novartis ahead of rivals like Kite Pharmaceuticals and on track to meet its lofty expectations for the treatment.

"In those settings, the downstream effect [of triggering a secondary cancer] is conceivable", said Maziarz.

And Wednesday's victory for Novartis, however incremental, buoyed the whole CAR-T field.

First developed by the University of Pennsylvania, CTL019 uses the 4-1BB co-stimulatory domain in its auto to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients. The drug maker cautioned that more work is needed.

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Contrary to expectations, the ODAC deliberations focused on relatively minor concerns about the manufacturing of CTL019 and the need to keep tabs on the data with the therapy after approval, and did not dwell for too long on the safety of the approach.

If Novartis can claim approval in ALL and follow-up indications such as diffuse large B-cell lymphoma (DLBCL), analysts reckon CTL019 could become a $1bn-plus product. Another risk is that there are many other players that are developing treatments in the CAR-T space as well. Millions of copies of the new T cells are grown in the lab and then injected into the patient's bloodstream where they can seek out and destroy cancer cells.

Such a complex system for making personalized treatments is likely to drive up their cost, and the next big hurdle (assuming an FDA approval this fall) is to win over insurers.

Each patient would have a copy of the drug suiting their body, their cells used in the manufacturing of their own drug.

The new drug class, known as chimeric antigen receptor T-cell or CAR-T, involves a complicated process of extracting immune system T-cells from an individual patient, altering the cell DNA to sharpen their ability to spot and kill cancer cells, and infusing them back into the patient. "Either delays or failure of production will have clinical consequences for the highest risk patients".

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