The FDA could be on the verge of approve the first gene therapy, after that an FDA advisory committee will decide on Wednesday 12th July whether to recommend approval of the approach, which uses patients’ own genetically altered immune cells to fight blood cancers. The panel of advisors are asked to focus on the safety of Novartis AG’s experimental gene therapy drug and vote on whether the benefits exceed the risks. Even if FDA is not obliged to follow the recommendations of its advisors, normally it does and in this case, tisagenlecleucel, would be the first gene therapy to be approved in the United States.
The FDA it is not asking the panel to focus on whether the drug works, as it successfully met the main goal of the clinical trial with 83 percent of patients who had relapsed or failed chemotherapy achieved complete or partial remission three months post infusion.
The excitement among doctors and researchers is palpable. However, with the enthusiasm come also pressing questions about safety, cost and the complexity of the procedure.
White blood cells called T cells are extracted from a patient’s blood, frozen and sent to Novartis’s plant in Morris Plains, N.J. There, a crippled HIV fragment is used to genetically modify the T cells so they can find and attack the cancer. The cells then are refrozen and sent back to be infused into the patient. Once inside the person’s body, the T-cell army multiplies astronomically.
Novartis hasn’t disclosed the possible price yet, but analysts expect to cost between $300,000 to $600,000 for a one-time infusion
The panel will be asked only to focus on the short-term and long-term safety risks. One of the most common side effect is cytokine release syndrome (CRS) which occurs when the body’s immune system goes into overdrive and cause high fever and flulike symptoms. Doctors could manage the condition and it caused no patient deaths. The other safety concern is neurotoxicity is which can result in temporary confusion or potentially fatal brain swelling. However, Novartis has not seen brain swelling in its trials, company officials said.
The FDA also raised concerns that the drug may cause secondary malignancies to occur and said long-term safety monitoring may be needed to address this concern.
Novartis is applying for approval in the first instance to treat B-cell acute lymphoblastic leukemia (ALL), the most common type of childhood cancer in the United States. However, the panel’s decision can have an impact also on Also Kite Pharma Inc. Juno Therapeutics Inc and bluebird bio Inc.
For instance, Kite Pharma of Santa Monica, Calif., has applied for FDA approval for aggressive non-Hodgkin lymphoma.
Novartis is also testing its drug in diffuse large b-cell Lymphoma (DLBCL), the most common form of non-Hodgkin lymphoma,
Part of the competitive landscape will include which company is best able to manufacture its drugs efficiently and reliably
UPDATES: Read about the FDA panel decision