It is the first gene therapy to be cleared in the United States – a “Historic” action, the agency said – and one with major ramifications for patients with cancer as well as other diseases.
“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.
The Novartis therapy is approved for patients who are up to 25 years old and have B-cell acute lymphoblastic leukemia that doesn’t respond to treatment or that returns after initial therapy – as happens in up to 20 percent of patients, according to the FDA. Novartis told reporters the treatment would cost $475,000; that makes it one of the most expensive of the new, high-priced oncology therapies.
In a pivotal study of more than five dozen patients, researchers reported, more than 80 percent of those who got the therapy went into remission. California-based Kite Pharma, which is being acquired by Gilead Sciences, also is seeking FDA approval for its CAR T-cell therapy for lymphoma patients. Gilead’s $11.9 billion purchase of groundbreaking cancer therapy could drag it into a new debate on prices.
“First gene therapy -“a true living drug’ – on the cusp of FDA approval.