The treatment is made by Novartis and is called Kymriah (tisagenlecleucel).
This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.
“This marks the first ever CAR-T cell therapy to be approved anywhere in the world,” Novartis CEO Joseph Jimenez told reporters on a conference call.
“It uses a new approach that is wholly personalized by using a patient’s own T-cells.”
Kymriah was approved by the US Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukemia (ALL).
To qualify for treatment, patients must have B-cell precursor ALL that is refractory, or the patient has relapsed at least twice.
The FDA described the approval as “a historic action” that would usher “in a new approach to the treatment of cancer and other serious and life-threatening diseases,” said a statement.
The treatment is not a pill or a form of chemotherapy, which can weaken the body’s natural defenses.
Instead, it harnesses a patient’s own immune cells, called T-cells and white blood cells, to recognize and fight cancer.
The patient’s immune cells are removed with a special blood filtration process, sent to a lab, and genetically encoded with a viral vector to be able to hunt down cancer cells.
These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukemia.
Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months, Novartis said.
An application with the European Medicines Association is expected to be filed by the end of the year.
– Rollout, cost –
The price of Kymriah — which is delivered to a patient just once — is $475,000, said Bruno Strigini, CEO of Oncology at Novartis.
Patients who do not respond to the treatment within the first month would not be expected to pay, he told reporters.
The more common treatment for leukemia — bone marrow transplants — can cost between $540,000 and $800,000 the first year in the United States, Stringing said.
Meanwhile, outside analyses set a cost-effective price for Kymriah between $600,000 and $750,000.
“Recognizing our responsibility we set the price below that level,” said Strigini.
Most patients who fit the criteria for treatment would likely be covered by insurance, since they are under 25 and would either be on their parents’ insurance or covered by Medicaid, a Novartis spokesman said.
– Breakthrough therapy –
The treatment was pioneered by Carl June at the University of Pennsylvania.
Its most high-profile patient is Emily Whitehead, now 12, who six years ago was the first child to receive what was widely considered a risky treatment.
She has been cancer free ever since.
In 2014, US regulators designated CTL019 as a “breakthrough therapy” and put the experimental immunotherapy agent on the fast track to market approval.
It was the first cancer immunotherapy to receive the breakthrough designation. More are expected to follow in the coming years as the field of immunotherapy grows.
Last month, an advisory panel to the FDA unanimously urged approval of the cutting edge cancer therapy.
About two dozen centers in the United States are expected to be able to treat patients seeking Kymriah, with as many as 35 up and running by the end of the year.
About 600 patients in the United States meet the criteria of being under 25 and having relapsed or refractory ALL, according to Novartis.