The FDA's Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favour of approving Novartis' ground-breaking CAR-T therapy for acute lymphoblastic leukaemia (ALL) yesterday, setting up a possible U.S. approval in September. The dramatic effect of the treatment, known for years as CTL-019, was never questioned at the meeting.
In a clinical trial, 79 percent of patients given the Novartis therapy were alive a year later.
Explaining their vote, many advisors were effusive.
"I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive", he added.
Novartis said the entire process would take 22 days by the time it is launched.
A breakthrough cancer therapy that can wipe out leukemia in some deathly ill children and young adults comes with an expensive, long-lasting side effect.
The drug enables patients' own immune cells to recognize and kill the source of the cancer: a different immune cell gone awry. The researchers plan to follow each patient for 15 years and will release information on 5-year survival rates when it becomes available. The promise of CAR-T cell therapy is evident not only in pediatric r/r ALL, but also across B Cell Malignancies, including adult ALL, aggressive r/r Non Hodgkin Lymphoma, and chronic lymphocytic leukemia.
Novartis' CTL019 is the first of many developing CAR-T therapies, which have put FDA in the unique position in which it has begun to monitor the safety events across more than 100 INDs. "You have to be a long-term survivor to experience [long-term] toxicity", said Bruce Roth of the Washington University School of Medicine in St. Louis, MO. "Our children deserve this chance", she said.
Novartis said that it has believed that CAR-T could "change the cancer treatment paradigm".
Nothing was helping until an experimental therapy called CAR-T.
Belldegren said he had been "amused" by the "horse race metaphors" used to describe the companies' attempts to get their therapies to market.
Shares of Novartis closed up 1.5% in Wednesday trading.
The FDA is expected to decide whether to approve the Novartis treatment in the next few months.
Many studies and trials have been conducted on the living drug, most of which have yielded positive results. Another company in the space, Juno Therapeutics, was forced to shut down its once-lead clinical candidate due to five deaths from cerebral edema - triggering concerns at the time about the class as a whole. Once reprogrammed the T cells are infused back into the patient in order to fight the cancer.
Such a complex system for making personalized treatments is likely to drive up their cost, and the next big hurdle (assuming an FDA approval this fall) is to win over insurers.
Novartis' tisagenlecleucel-T, first developed by scientists at the University of Pennsylvania, is made by taking blood from cancer patients, separating out T cells and inserting in them a chimeric antigen receptor (CAR) that targets CD19, which is an antigen that's expressed on some blood cancers. These are in patients that have failed everything.