publication date: Sep. 1, 2017

Drugs and Targets

Novartis’s Kymriah becomes first gene therapy to get FDA approval

Kymriah (tisagenlecleucel) became the first gene therapy available in the United States.

The agent, sponsored by Novartis Pharmaceuticals Corp., was approved for pediatric and young adult patients with a form of acute lymphoblastic leukemia.

On July 12, the FDA Oncologic Drugs Advisory Committee unanimously recommended that the agent be approved (The Cancer Letter, July 14).

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a statement announcing the agent’s approval. “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. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”

Kymriah, a cell-based gene therapy, received approval for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second- or later relapse.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a chimeric antigen receptor—CAR—that directs the T-cells to target and kill leukemia cells that have a specific antigen—CD19—on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

NCI estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year. ALL can be of either T- or B-cell origin, … Continue reading Novartis’s Kymriah becomes first gene therapy to get FDA approval

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