The U.S. Food and Drug Administration Wednesday approved Novartis’ CAR-T treatment Kymriah (ingredient: tisagenlecleucel) to fight rare childhood leukemia, ushering in a new approach to cancer treatment, the state agency said.
Kymriah is the first-ever customized cell treatment that uses a patient’s T-cells to fight cancer.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack deadly cancer,” said FDA Commissioner Scott Gottlieb 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.”
Each dose of Kymriah is created by collecting the patient’s T-cells and genetically modifying them at a manufacturing center to include a new gene with a chimeric antigen receptor (CAR), a protein that directs T-cells to kill leukemia cells with a specific antigen (CD19).
A multicenter clinical trial -- involving 63 pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) -- proved the drug’s safety and efficacy. The overall remission rate within three months of treatment was 83 percent, the company said.
Doctors may prescribe the drug to patients up to 25 years of age diagnosed with B-cell precursor ALL that is refractory or in second or later relapse, the state agency said.
ALL is a bone marrow and blood cancer that produces abnormal lymphocytes. The illness is the most common cancer for children in the U.S. with the National Cancer Institute estimating around 3,100 patients less than 20 years of age diagnosed each year.
“Kymriah is a first-of-its-kind treatment approach that fills a critical unmet need for children and young adults with this serious disease,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. “Not only does Kymriah provide these patients with a new treatment option where insufficient options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”
The FDA approval marks a victory for Novartis as it crossed the figurative finish line ahead of lead rival Kite Pharma, which is also developing CAR-T therapies. Both made progress this year with Novartis gaining a unanimous FDA panel vote favoring approval for the drug and Gilead Science’s acquiring Kite Pharma for $11.9 billion Monday. Kite Pharma’s CAR-T drug Axi-Cel is currently under priority review by the FDA with results to come out in November.
■ Related : Gilead to acquire Kite Pharma for $11.9 billion
Novartis has reportedly unveiled a price of $475,000 for a one-time treatment, effectively becoming one of the most expensive drugs to date.
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