EC Approves Novartis’ Kymriah CAR-T Cell Therapy

On Aug. 27, 2018, Novartis announced that the European Commission (EC) approved its chimeric antigen receptor T cell (CAR-T) cell therapyKymriah(tisagenlecleucel) for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. 

Kymriah, a cell dispersion for infusion with doses varying between 1.2 x 10⁶, 6 x 10⁸ CAR- positive viable T cells, is a living medicinal product manufactured individually for each patient by reprogramming the patient's own immune system cells. According to Novartis, Kymriah is built using the 4-1BB costimulatory domain, which is critical for full activation of the therapy, advancement of cellular expansion, and durable persistence of the cancer-fighting cells. 

Kymriah was designated as an orphan medicinal product and is one of the first PRIority MEdicines (PRIME)-designated therapies to receive approval in the European Union (EU). The treatment was developed in collaboration with the University of Pennsylvania (Penn) as a one-time treatment and is the only CAR-T therapy to receive regulatory approval in the EU for these two distinct B-cell malignancies, as stated by Novartis. In 2012, Novartis and Penn entered into a global collaboration to further research, develop, and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers. 

Kymriah was approved by FDA in August 2017 for the treatment of ALL and in May 2018 for the treatment of adult patients with r/r DLBCL after two or more lines of systemic therapy.

Both B-cell ALL and DLBCL are aggressive malignancies with significant treatment gaps for patients. Initially, the company expects to launch the therapy in the pediatric ALL indication as capacity increases. Additionally, timing for Kymriah availability in each country will depend on multiple factors, including the onboarding of qualified treatment centers for the appropriate indications, as well as the completion of national reimbursement procedures. Training is already underway at key qualified treatment centers to facilitate safe delivery to patients.

Novartis also has been actively pursuing options to expand manufacturing capabilities beyond its facility in Morris Plains, NJ. This includes the company’s agreement with France-based, contract development and manufacturing organization (CDMO) CELLforCURE to produce cell and gene therapies in Europe, the expanded alliance with Fraunhofer Institute, which currently supports the manufacturing of Kymriah for global clinical trials and for post approval manufacturing, as well as technology transfer efforts to a CDMO in Japan.

Source: Novartis