Novartis CAR-T Cell Therapy CTL019 Recommended for FDA Approval

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FDA advisory committee has recommended for approval Novartis CAR-T cell therapy CTL019 for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia in pediatric and young adult patients.

On July 12, 2017, Novartis announced that FDA’s Oncologic Drugs Advisory Committee (ODAC) has unanimously recommended approval of CTL019 (tisagenlecleucel) for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). The ODAC recommendation is based on review of the CTL019 r/r B-cell ALL development program, which includes the ELIANA study (NCT02435849), led by Novartis. This study represents the first pediatric global CAR-T cell therapy registration trial. Findings from a US multicenter trial and a single-site trial evaluating the safety and efficacy of CTL019 in pediatric and young adult patients with r/r B-cell ALL also supported the recommendation and the biologics license application (BLA). 

CTL019 is an investigational chimeric antigen receptor T cell (CAR-T) therapy, first developed by the University of Pennsylvania (Penn). It uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular responses and persistence of CTL019 after infusion into the patient, which is believed to provide long-lasting remissions in patients. 

In 2012, Novartis and Penn entered into a global collaboration to further research, develop, and commercialize CAR-T cell therapies, including CTL019, for the treatment of cancers. Children’s Hospital of Philadelphia (CHOP) was the first institution to investigate CTL019 in the treatment of pediatric patients and led the single-site trial.



Novartis submitted a BLA for CTL019 to FDA in early 2017, marking the first submission by Novartis for a CAR-T cell therapy. CTL019 has been given breakthrough therapy designation by FDA and is under priority review. FDA will consider the vote as it reviews the BLA, but is not obligated to follow the recommendation. Novartis continues to invest in the necessary infrastructure for the potential commercialization of CTL019, including manufacturing and the establishment of a network of certified treatment centers.

Novartis plans additional filings for CTL019 in the United States and the European Union later in 2017, including applications with FDA and the European Medicines Agency for the treatment of adults with r/r diffuse large B-cell lymphoma.

Source: Novartis