Novartis AG's pioneering cancer drug won the backing of a federal advisory panel Wednesday, paving the way for the first gene therapy to be approved in the United States.
An advisory panel to the Food and Drug Administration 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 U.S. 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 $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' drug showed that 83 percent 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 a 16 to 30 percent 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.
More than half of patients experienced a serious complication known as cytokine release syndrome (CRS), which occurs when the body's immune system goes into overdrive.
Doctors were able to manage the condition, and the syndrome caused no deaths.
The FDA expressed concern that the drug could cause new malignancies over the long term, but panelists generally felt that risk was low.