Novartis files for European approval of CAR-T cell therapy CTL019 in two indications

Novartis announced Monday the submission of a marketing application to the European Medicines Agency seeking approval of CTL019 (tisagenlecleucel) for two indications, including for the treatment of children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL). The filing also seeks clearance of the CAR-T cell therapy for adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant.

In August, CTL019 became the first available CAR-T cell therapy when it received approval in the US, where it is marketed as Kymriah, for patients up to 25 years of age with B-cell precursor ALL that is refractory or has relapsed at least twice. Meanwhile, last month, Novartis submitted a filing to the FDA seeking approval of Kymriah for the treatment of adults with relapsed or refractory DLBCL who are ineligible for autologous stem cell transplant.

According to Novartis, the submission in Europe is based on findings from the mid-stage ELIANA and JULIET trials. Data from the six-month primary analysis of JULIET will be presented at the annual meeting of the American Society of Hematology (ASH) next month. The company is planning further regulatory filings in the two indications outside the US and EU in 2018.

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CTL019, which is given as a one-time treatment, uses the 4-1BB costimulatory domain in its chimaeric antigen receptor to enhance cellular expansion and persistence. In 2012, Novartis and the University of Pennsylvania entered into a global collaboration to develop CAR-T cell therapies, including CTL019.

Last month, the FDA approved Gilead Sciences' CAR-T therapy Yescarta (axicabtagene ciloleucel) for adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic treatment, including DLBCL not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma and DLBCL arising from follicular lymphoma. A regulatory filing for Yescarta as a treatment for patients with relapsed/refractory DLBCL, transformed follicular lymphoma and primary mediastinal B-cell lymphoma who are ineligible for autologous stem-cell transplant is currently under review by the EMA, with a potential approval expected in the first half of 2018.

For related analysis, see ViewPoints: Novartis filing, ASH abstracts bring CAR-T competition a step closer.

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