Wednesday, 22 November, 2017

Novartis approval for gene therapy signals new cancer treatment era

The logo of Swiss drugmaker Novartis AG is seen at its headquarters in Basel Switzerland The logo of Swiss drugmaker Novartis AG is seen at its headquarters in Basel Switzerland
Sammy Stanley | 31 August, 2017, 01:23

The U.S. Food and Drug Administration on Wednesday approved Novartis AG's (NVS) Kymriah, a cell-based gene therapy for certain children and young adults with B-cell acute lymphoblastic leukemia.

"Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials", said Peter Marks, MD, PhD, director of the FDA.

Its approval is likely to set the stage for the FDA to approve a slew of CAR-T drugs over the next several years.

This article will be updated.

At the time, American Cancer Society Deputy Chief Medical Officer Dr. Len Lichtenfeld said the real promise of these gene therapies is the potential to completely eradicate specific cancers both for the single patient as well as all their descendants. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses".

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus.

But he and other researchers say thousands more patients eventually may benefit.

Returned to the patient, the cells can continue multiplying to fight disease for months or years.

"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement. This therapy - known as chimeric antigen receptor T-cell therapy, or CAR-T - gives the cells the ability to recognize and kill the source of the cancer.

Because patients can develop life-threatening side effects weeks after the procedure, doctors will ask patients to stay within two hours of the hospital for up to a month. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.

Because of these safety concerns, the FDA will require that hospitals receive special certification to use Kymriah, the agency said.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CRS in patients 2 and older.

For some patients, the new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis study. Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year.

The FDA also is considering a auto T-cell therapy from California-based Kite Pharma.

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