FDA advisers recommend approval of new cancer therapy

The FDA panel made their recommendation after reviewing CTL019's performance in a range of clinical trials including the ELIANA study, which was the first pediatric global CAR-T cell therapy registration trial.

This advisory committee hearing was the last major regulatory milestone before the agency decides in September whether or not to approve the treatment, which would make Novartis' CAR-T therapy the first-ever gene therapy treatment approved by the FDA in U.S. markets.

The treatment, called CTL019, genetically tweaks a patient's own immune system cells into what scientists call "a living drug" to battle a form of acute lymphoblastic leukemia (ALL), The New York Times reported. Though the approval wouldn't necessarily be surprising in light of the results of the trials, which were stunning: patients facing death after all other treatments failed who received just a single dose of the gene therapy experienced long remissions that could, in time, prove curative. The FDA doesn't have to follow the advice of the advisory committee, but it often does.

Gaining approval for CTL019 could make it the first officially approved gene therapy in the United States.

"We're very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options".

This revolutionary new method of treating blood cancer is customized to target a specific type of cancer, that is, it is adjusted according to the DNA information of a particular patient.

The treatments involve extracting white blood cells or T-cells which defend the immune system, from cancer patients.

"While CAR-T is a promising new type of immunotherapy, it is not commercially available and we have yet to complete our evaluation", T.J. Crawford, a spokesman for health insurer Aetna Inc said in a statement. Kite Pharma of Santa Monica, Calif., has applied for FDA approval to treat aggressive non-Hodgkin's lymphoma, and a similar Novartis application is close behind. The patient numbers were small, but the responses were extraordinary for the patients who responded, with an 83% overall remission rate and 79% overall survivability rate at 12 months - hitting the primary endpoint. Once reprogrammed the T cells are infused back into the patient in order to fight the cancer.

Novartis' risk mitigation also includes an education program for the sites providing the treatment and a registry, which the ODAC members and the FDA cited as essential to tracking long-term risks and better understanding of T cells.

The treatment, called CTL019, has been developed by Swiss pharmaceutical major Novartis.

Each treatment is created for each individual patient by having their cells removed, frozen and shipped to a Novartis plant for processing. The ruling on the use of the CAR-T gene therapy will be announced by the end of September. But treatment gave their daughter a few extra months of happy life, Kappen said.

Meanwhile, the FDA expressed concern over the side effects of the drug.

CAR-Ts can also pose serious risks, including a potentially life-threatening inflammatory condition. ODAC members expressed satisfaction with Novartis' plan to minimize risk, which includes limiting distribution of the therapy to selected centers experienced with auto T-cell therapy (at least initially) and plans for extensive, long-term postmarketing surveillance.

  • Aubrey Nash