Regulatory Affairs

Novartis seeks EMA approval for CTL019 for two indications

PBR Staff Writer Published 07 November 2017

Novartis is seeking approval from the European Medicines Agency (EMA) for its CTL019 (Kymriah or tisagenlecleucel) to treat two forms of blood cancer.

The company has submitted marketing authorization application (MAA) to the EMA for Kymriah to treat children and young adults with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (ALL), as well as adult patients with r/r diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant (ASCT).

CTL019 is a novel immunocellular therapy and a one-time treatment, which uses a patient's own T cells for the treatment of cancer.

It uses the -1BB costimulatory domain in its chimeric antigen receptor to increase cellular expansion and persistence.

In 2012, Novartis collaborated with the University of Pennsylvania (Penn) to further research, develop and commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers.

The company has submitted the application based on the data of global, multicenter and phase II Eliana and Juliet trials, which were carried out in collaboration with Penn.

Eliana is said to be the first pediatric global CAR-T cell therapy registration trial, which is assessing patients in 25 centers in the US, Canada, Australia, Japan and the EU countries such as Austria, Belgium, France, Germany, Italy Norway, and Spain.

Juliet is the first multi-center global registration study for CTL019 in adult patients with r/r DLBCL.

It is evaluating a CAR-T therapy exclusively in patients with DLBCL at 7 sites in 10 countries across the US, Canada, Australia, Japan and European countries such as Austria, France, Germany, Italy, Norway and the Netherlands.

Novartis chief medical officer and drug development global head Vas Narasimhan said: "Since the historic FDA approval of Kymriah, formerly CTL019, we have launched, manufactured and supplied this highly individualized immunocelluar therapy in a commercial setting and the submission to the EMA is a major step toward our goal of delivering it to more critically ill cancer patients around the world.”