Novartis (NVS) : FDA approves first gene therapy; costing $475,000

 The Food and Drug Administration approved Novartis’s gene therapy for leukemia, the first-ever treatment that alters a patient’s own cells to fight cancer.

• The gene cell therapy involves drawing blood from children with B-cell acute lymphoblastic leukemia. 
• The white blood cells called T-cells in the child's blood are then shipped to Novartis in Morris Plains, N.J.  where they are genetically engineered so they will seek out a particular protein in the leukemia cells and attack. 
• They will be frozen again and shipped back to the medical center to be dripped into the patient. That processing is expected to take 22 days.
• Patients are then infused with the modified blood, and the T-cells go to work to find and kill the leukemia. 
• The Kymriah  therapy will cost $475,000 for the initial treatment, with additional treatments administered at no cost. 
• In the study that led to Kymriah’s approval, 83 percent of patients went into remission within three months, according to the FDA. Novartis estimates about 600 patients a year would be eligible for the treatment, which belongs to a class of drugs known as CAR T-cell therapies.
• Novartis also considered the cost of bone-marrow transplants, which are currently given to many leukemia patients whose cancer relapses. Those transplants can cost up to $800,000, Novartis said.
• The FDA also is considering a CAR T-cell therapy from California-based Kite Pharma. Gilead Sciences — which has been criticized for the $84,000 price tag of its hepatitis C drug — recently announced that it will buy Kite for $11.9 billion.

The Food and Drug Administration on Aug. 30, 2017 approved the first-ever treatment that genetically alters a patient’s own cells to fight cancer, a milestone that is expected to transform treatment in the coming years.

The new therapy turns a patient’s cells into a “living drug,” and trains them to recognize and attack the disease. It is part of the rapidly growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long remissions and possibly even cures.

A technician working with human cells belonging to cancer patients at Novartis Pharmaceuticals in Morris Plains, N.J.

The therapy, marketed as Kymriah and made by Novartis, was approved for children and young adults for an aggressive type of leukemia — B-cell acute lymphoblastic leukemia — that has resisted standard treatment or relapsed. The FDA called the disease “devastating and deadly” and said the new treatment fills an “unmet need.”

Novartis and other companies have been racing to develop gene therapies for other types of cancers, and experts expect more approvals in the near future. Dr. Scott Gottlieb, the FDA commissioner, said that more than 550 types of experimental gene therapy were being studied.

There are drawbacks to the approach. Because Kymriah can have life-threatening side effects, including dangerous drops in blood pressure, the FDA is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.

Kymriah, which will be given to patients just once and must be made individually for each, will cost $475,000. Novartis said that if a patient does not respond within the first month after treatment, there will be no charge. The company also said it would provide financial help to families who were uninsured or underinsured.

Discussing the high price during a telephone news conference, a Novartis official noted that bone-marrow transplants, which can cure some cases of leukemia, cost even more, from $540,000 to $800,000.

About 600 children and young adults a year in the United States would be candidates for the new treatment.

The approval was based largely on a trial in 63 severely ill children and young adults who had a remission rate of 83 percent within three months — a high rate, given that relapsed or treatment-resistant disease is often quickly fatal.

The treatment was originally developed by researchers at the University of Pennsylvania and licensed to Novartis. It was identified in previous reports as CAR-T cell therapy, CTL019 or tisagenlecleucel.