Regulatory Affairs

FDA advisory committee recommends Novartis’ CTL019 for r/r B-cell ALL

PBR Staff Writer Published 13 July 2017

The US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) has recommended Novartis’ CTL019 (tisagenlecleucel) to treat relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).

The CTL019 is an investigational chimeric antigen receptor T cell (CAR-T) therapy developed to treat patients with r/r B-cell ALL.

The recommendation of ODAC was based on the evaluation of the CTL019 r/r B-cell ALL development program that included the firm-lead ELIANA study, which is claimed to be the first pediatric global CAR-T cell therapy registration trial.

Data from the US multicenter trial and single site trial assessing the safety and efficacy of CTL019 among pediatric and young adult patients with r/r B-cell ALL also backed the recommendation and the biologics license application (BLA).

The University of Pennsylvania (Penn) has first developed CTL019, which uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular responses.

In 2012, Novartis and Penn signed a collaboration to further research, develop and commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers.

At the staring of this year, Novartis filed a BLA for CTL019 to the FDA. The CTL019 also secured FDA breakthrough therapy status and is under priority review by the FDA.

In addition, the firm is planning additional filings for CTL019 in the US and EU later this year, including applications with the FDA and European Medicines Agency (EMA) to treat adults with r/r diffuse large B-cell lymphoma (DLBCL).

Novartis Oncology CEO Bruno Strigini said: "The panel's unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need.

"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.”

Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis AG.