One year on: Kymriah® CAR-T cell therapy in Germany
- Around one year ago, the European Commission approved the use of Novartis’ CAR-T cells in Germany for patients diagnosed with certain types of life-threatening blood cancer who have extremely limited survival prospects.
- Each patient-specific CAR-T cell infusion is the result of an individualized, innovative manufacturing and logistics process that Novartis has established together with the Fraunhofer Institute for Cell Therapy and Immunology IZI in Leipzig.
- In Germany, more than 15 hospitals1 are now certified to carry out the CAR-T cell therapy developed by Novartis.
Leipzig / Nuremberg, 07/10/2019 – Just over a year ago, on 27 August 2018, the European Commission gave their approval for the CAR-T cells developed by Novartis to be used in Germany. Since then, patients with certain types of life-threatening blood cancer and extremely limited survival prospects may, for the first time, have an opportunity to prolong their life thanks to the CAR-T cell therapy developed by Novartis. In Germany, more than 15 hospitals1 are now certified to carry out this treatment. Novartis worked in close collaboration with the Fraunhofer Institute for Cell Therapy and Immunology IZI to establish a complex process for CAR-T cells.
The CAR-T cell therapy is an immune-enhancing therapy based on T cells taken from the treated patient’s own body. The patient’s T cells are recruited and genetically modified to feature a special immunocompetence that is able to fight cancer cells. After reintegrating the CAR-T cells in the immune system, cancer cells can be recognized in the body and destroyed effectively. The therapy is therefore not at all reliant on donors and can be carried out even if the patient is not in complete remission.
Patients suffering from two rare life-threatening illnesses can benefit from this therapy2:
- children and young adults up to 25 years old with relapsed or refractory acute lymphoblastic leukemia (r/r pALL);
- adults with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL).
"The CAR-T cell therapy marks a small revolution in hematology," says Prof. Uwe Platzbecker. He is the director of Medical Clinic I, Hematology and Cell Therapy at Leipzig University Hospital. Novartis worked closely together with the German sites even during the pivotal trial phase.
Patient-specific CAR-T cell infusions
Each patient-specific CAR-T cell infusion is the result of an individualized, innovative manufacturing and logistics process. Working together with the renowned Fraunhofer Institute for Cell Therapy and Immunology IZI in Leipzig, Novartis established this novel manufacturing process for CAR-T cells also in Germany. "With Kymriah®, cell and gene therapy has now found its place in patient care and will establish itself as an additional pillar of cancer treatment in the years to come. As a research institute, we strive to work with our partners to open up new areas of application and to optimize complex process flows," remarks Prof. Ulrike Köhl, Director of Fraunhofer IZI. "The 21 clean-room suites at our Leipzig site are used by over 80 qualified members of staff to produce a living drug that takes effect over a long period of time. Extensive quality controls at every stage of production ensure the high quality of the patient-specific preparations," adds Dr. Gerno Schmiedeknecht, Head of the Main Department of Cell and Gene Therapy at Fraunhofer IZI.
More than 15 hospitals1 are providing CAR-T cell therapy
Novartis is continuously working on building up a close-meshed network of highly qualified treatment centers. In Germany, more than 15 hospitals1 have been certified to date to carry out the CAR-T cell therapy developed by Novartis. These centers meet exacting criteria such as profound expertise in the indication, comprehensive knowledge of the processes involved in complex cell therapies, e.g. stem cell transplants, and the availability of facilities needed to manage possible side effects.
Since 15 September 2019: reimbursement amount agreed for CAR-T cell therapy
On 15 September 2019, the National Association of Statutory Health Insurance Funds and Novartis agreed on a reimbursement amount for the CAR-T cell therapy. This sum takes into consideration the high level of innovation and the therapy’s clinical value for patients with DLBCL and pALL, with an eye to cost efficiency aspects. "We are pleased to have come to an agreement on a reimbursement amount for the CAR-T cell therapy as the result of open and constructive talks with the National Association of Statutory Health Insurance Funds," says Markus Karmasin, Head of Cell and Gene Therapy at Novartis Oncology in Germany, Novartis Pharma GmbH. "The conclusion of both the price negotiations between the National Association of Statutory Health Insurance Funds and Novartis as well as the negotiation of fees for new examination and treatment methods between the hospitals and health insurance funds means we can now focus our attention on patient care."
CAR-T cell therapy is one of four innovative approaches to cancer therapy. Novartis is the only company to utilize all four therapeutic platforms: targeted therapies, immuno-oncology, immune-enhancing cell therapy and radionuclide therapy. This ties in with Novartis’ aim to reimagine cancer through the combination of these innovative treatment strategies and thus revolutionize therapy – for some forms of cancer at least.
Further information on CAR-T cell therapy can be found at:
About CAR-T cell therapy3, 4
CAR-T cell therapy is a new type of cancer immunotherapy that uses the patient’s own T cells to fight certain types of cancer. In order to do this, the cells are extracted by leukapheresis and then genetically reprogrammed in vitro in such a way that they can use their chimeric antigen receptor to recognize cancer cells and other cells that have a specific antigen on their surface. Following lymphodepleting chemotherapy, the reprogrammed cells are fed back to the patient though an infusion. The cells then multiply and can trigger the immune response.
About Kymriah® (tisagenlecleucel)2
In August 2017, the first CAR (chimeric antigen receptor)-T cell therapy became available in the USA in the form of Kymriah® (tisagenlecleucel). Kymriah® was granted FDA approval for children and young adults up to the age of 25 years who have acute lymphocytic B-cell leukemia that is therapy-refractory or has relapsed at least twice. Approval was subsequently granted in May 2018 for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. The therapy is an innovative one-time immunotherapy which uses the patient’s own genetically modified cells in the fight against cancer cells. Kymriah® uses the 4-1BB costimulatory domain as part of the chimeric antigen receptor in order to increase the expansion and persistence of the cells.
Novartis seeks to find new ways to help people live a longer and better life. As a leading global pharmaceutical company, we use scientific innovations and digital technologies to develop breakthrough therapies in areas of high medical need. In doing so, we regularly count among the companies that invest the most in research and development around the world. Novartis' products reach more than 750 million people worldwide and we are looking for new ways to expand access to our latest treatments. Novartis employs approximately 105,000 people from more than 140 countries around the world. Further information can be found online at http://www.novartis.com.
1 Effective: September 2019
2 Novartis Pharma, prescribing information (summary of product characteristics/SmPC), Kymriah® 1.2 × 106 to 6 × 108 cells for infusion dispersion, effective: September 2018
3 Abken H. CAR-T Zellen: Wie Designer-Immunzellen gegen den Krebs scharfgemacht werden können. Trillium Immunologie 2017; 1(1):43-47.
4 Tran E et al. A Milestone for CAR T Cells. New Engl J Med 2017. DOI: 10.1056/NEJMe1714680.
Head External Affairs, Patient Relations & Communications Oncology
Novartis Pharma GmbH
Phone +49 911 273 12940