FDA Approves First Gene Therapy


Why the agency says the approval of Novartis’ new therapy is historic.

The FDA announced today that it has granted approval to the first ever gene therapy available in the United States. The administration called the approval of Novartis’ new therapy “historic,” saying that the approval “usher[ed] in a new approach to the treatment of cancer and other serious and life-threatening diseases.”

Kymriah (tisagenlecleucel), is a customized, genetically-modified autologous T-cell immunotherapy for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL).  It is the first FDA-approved chimeric antigen receptor T cell (CAR-T) therapy. The treatment utilizes a patient’s T-cells to create a personalized treatment. Those T-cells will be sent to a New Jersey-based Novartis facility, where the manufacturer will then modify the cells to attack leukemia cells.

“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, MD in a statement. “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.”

ALL is the most common cancer in children in the United States. Each year, around 3,100 patients age 20 or younger are diagnosed with ALL. While ALL can be of either T- or B-cell origin, B-cell is the most common.

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In a clinical trial at multiple sites, 63 pediatric and young adults with elapsed or refractory B-cell precursor ALL were given the treatment. Within three months, 83% of patients achieved complete remission. No minimal residual disease (MRD), a blood marker that can indicate the possibility of relapse, was detected in patients who responded to the treatment.

The treatment carries the possibility for severe side effects. It has a boxed warning for cytokine release syndrome (CRS), a response to the activation and proliferation of CAR-T cells, which causes high fever and flu-like symptoms. It also carries a warning for neurological events. The FDA is requiring hospitals that dispense Kymriah to be specially certified-which includes staff being trained to recognize and manage CRS and neurological events.

The FDA also approved a new indication for Genentech's Actemra (tocilizumab) for the treatment of CAR-T incuced CRS.

Kymriah was granted Fast Track, Priority Review, and Breakthrough Therapy designations. According to Fortune, the drug will have a cost of $475,000 for a one-time treatment, but will be subject to outcome-based pricing-for patients who don’t respond to treatment within the first month, Novartis will not be reimbursed by CMS. 

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