FDA panel unanimously backs approval of Novartis CAR

FDA advisers back Novartis' game-changing personalized CAR-T for cancer, approval in sight
FDA Panel Recommends Approval for Gene-Altering Leukemia Treatment

14 July, 2017

While this gene therapy treatment for leukemia, known as CTL019, will be the first to reach the market, there are more on the way.

The FDA's Oncologic Drugs Advisory Committee's recommendation of a leukemia treatment using gene therapy could be a boon to supporters of the technology, Dr. Carl June, leader of the experimental therapy, said in a statement Wednesday. The company said that in its clinical trials, it was able to detect CAR-T cells in patients who respond to therapy for as long as six months, with recovery of normal B cells taking from a few months to more than one year, said Christine Cassiano, a spokeswoman for the Santa Monica, California-based company. This process gives the patient's immune system a tumor-attack roadmap for the treatment of leukemia and other cancers, including those of the ovaries and myeloma. The FDA is not bound by the decisions of its advisory committees but follows the recommendations more often than not.

The Food and Drug Administration advisory panel has voted in favor of this new gene treatment on which the members voted 10-0.

This therapy is a one-time treatment for children and young adults affected by the most common form of childhood cancer, acute lymphoblastic leukemia or ALL.

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.

"Although this therapy is technologically somewhat complicated and is associated with certain serious side effects, it, indeed, has been proven to be amazingly effective", said Kanti Rai, chief of the chronic lymphocytic leukemia research and treatment program at Northwell Health Cancer Institute in NY.

Novartis' filing contains data - presented at the American Society of Haematology meeting in December 2016 - from a Phase II study in which 82 percent (41 of 50) of patients infused with CAR-T cells achieved complete remission or complete remission with incomplete blood count recovery at three months post CTL019 infusion.

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They included Tom Whitehead whose then 6 year old daughter, Emily, was the first to try CAR-T at Children's Hospital of Philadelphia.

He said in a blog: "With every paper I read, it becomes more and more clear that CAR-T is not a biotech race to be won, but rather a revolutionary therapy to be explored and applied to other cancers".

Scientists are calling the treatment "a living drug" as it harnesses the immune system to beat cancer, according to The New York Times.

Variations in quality attributes, potency, and transduction efficiency of Novartis' biological cell therapy CTL019, however, did not translate into changed safety or efficacy, according to statistical analyses. Most patients suffered serious side effects but almost all recovered.

In this pivotal study informing the committee's decision, roughly half of 68 patients receiving the drug experienced this complication, though none died from it. She got vehicle T-cell therapy when she was 6 and close to death from leukemia. To generate a batch of tisagenlecleucel, white blood cells are purified from a sample of a patient's blood and shipped to a central processing centre.

Last year, when Kite hired Christine Cassiano its new SVP of corporate communications and investor relations, she said that communication around CAR-T therapies is "about making it relatable to patients". The same goes for personalized immunotherapy: Dendreon's Provenge was FDA-approved to treat prostate cancer in 2010, for example.

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