The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the USA market - and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumours.
The treatment will be sold under the name Kymriah, and is approved for children and young adults up to age 25 with cancers that don't respond to traditional treatment, or who are suffering from a second relapse, a broad indication that will give more patients access to the novel technology. The treatment reprograms a patient's T cells to carry a "chimeric antigen receptor" that targets cancer 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", FDA Commissioner Scott Gottlieb said.
"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population", Dr Stephan Grupp of Children's Hospital of Philadelphia said in a statement. Within 3 months of treatment, the overall remission rate was 83%. According to Children's of Alabama, more than 190 children in Alabama are diagnosed with various forms of cancer each year, including ALL. The FDA also expanded approval of tocilizumab (Actemra) for the treatment of CAR-T cell-induced severe or life-threatening CRS in patients 2 years or older, according to the release.
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For more on childhood acute lymphoblastic leukemia, visit the U.S. "At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving", he said. Once modified, the cells are infused back into the patient to kill the cancer cells. Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year.
The company said it plans to work with the Centers for Medicare and Medicaid Services to implement the pricing strategy.
The one-time treatment has a boxed warning for cytokine release syndrome or CRS, a life-threatening side effect that can cause blood pressure to drop dangerously low. It also said that it is working with CMS on an outcomes-based approach to reimbursement that will allow payment for only those ALL patients that respond to treatment by the end of one month.
Novartis is required to conduct followup study to assess the safety of the treatment long-term.