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FDA Greenlights Novartis' Gene Therapy for Leukemia

31 August 2017, 01:44 | Lucy Hill

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

US clears first 'living drug' for childhood leukaemia

The treatment will be sold under the name Kymriah, and is approved for children and young adults up to age 25 with cancers that don't respond to traditional treatment, or who are suffering from a second relapse, a broad indication that will give more patients access to the novel technology.

Kymriah belongs to a new class of drugs called chimeric antigen receptor T-cell, or CAR-T therapies, that involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and fight cancer and then replacing them, where they can circulate for years seeking out the disease.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb, who was appointed to the position by President Donald Trump earlier this year. The company did say that it's made an agreement with the USA government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month. It transforms a patient's immune system cells to fight a form of leukemia. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte.

The National Cancer Institute estimates 3,100 patients are diagnosed each year with ALL, which can come in several forms. More than half were children and teens.

It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple.

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"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in a statement. "At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving", he said.

Treatment with Kymriah also has the potential for severe side effects, including cytokine release syndrome, which leads to high fever, flu-like symptoms, and other neurological events.

The company said it plans to work with the Centers for Medicare and Medicaid Services to implement the pricing strategy.

In a key study of 63 advanced patients, 83% went into remission, although it is not clear how long the benefit lasts: Some patients did relapse months later. The FDA also released a treatment for these side effects on Wednesday.



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