Novartis’ pioneering cancer gene therapy drug get US panel support

A pioneering cancer drug treatment developed by Swiss multinational pharmaceutical company Novartis AG has received support from an advisory panel to the US Food and Drug Administration (FDA), which could lead to the drug becoming the first gene therapy to be approved in the US.

The panel voted 10-0 that the drug, tisagenlecleucel, should be approved to treat patients with relapsed B-cell acute lymphoblastic leukemia (ALL), the most common form of US childhood cancer.

The FDA is not obliged to follow the recommendations of its advisers but typically does so. The agency is expected to rule on the drug by the end of September.

Approval of tisagenlecleucel would have significant implications not only for Novartis but for companies developing similar treatments, including Kite Pharma Inc., Juno Therapeutics Inc., and bluebird bio Inc.

All four are developing chimeric antigen receptor T-cell therapies (CAR-T), which harness the body’s own immune cells to recognize and attack malignant cells.

If approved, the drugs, which are infused just once, are expected to cost up to US$500,000 and generate billions of dollars for their developers. Success would also help advance a cancer-fighting technique that scientists have been trying to perfect for decades and lift the broader field of cell therapy.

“In the last five years there have been a significant number of cell therapy companies that have gone public or gotten investment in hopes of moving this type of therapy forward,” said Reni Benjamin, an analyst at Raymond James. “This is our first glimpse from a commercial and regulatory perspective about how the FDA is thinking about this space.”

A clinical trial of Novartis’s drug showed that 83% of patients who had relapsed or failed chemotherapy, achieved complete or partial remission three months post infusion. Patients with ALL who fail chemotherapy typically have only a 16% to 30% chance of survival.

Novartis is also testing the drug in diffuse large b-cell Lymphoma (DLBCL), the most common form of non-Hodgkin lymphoma, as is Kite. Part of the competitive landscape will include which company is best able to manufacture its product most efficiently and reliably.

The products are made by extracting and isolating a patient’s T cells, genetically engineering them to recognize and target specific cancer cells, and then infusing them back into the patient. Novartis said the entire process will take 22 days by the time it is launched.

A serious complication known as cytokine release syndrome (CRS), which occurs when the body’s immune system goes into overdrive, was experienced by more than half of the patients. Doctors were able to manage the condition and the syndrome caused no deaths.

The FDA also expressed concern that the drug could cause new malignancies over the long term, but panelists generally felt that risk was low.

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Category: Features, Pharmaceuticals