EU approves Novartis' CAR-T cell therapy Kymriah for blood cancers

Novartis announced Monday that the European Commission approved its filing for the CAR-T cell therapy Kymriah (tisagenlecleucel) for blood cancer indications. Specifically, Kymriah is cleared to treat paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. The CAR-T cell therapy, formerly known as CTL019, is also approved for the treatment of adults with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. 

The approval follows a positive recommendation by the European Medicines Agency's Committee for Medicinal Products for Human Use in June. The filing was backed by data from the Phase II ELIANA study of children and young adults with r/r B-cell ALL, as well as the JULIET trial in adults with r/r DLBCL. 

Liz Barrett, CEO of Novartis Oncology, remarked that "the Kymriah approval is a transformational milestone for patients in Europe in need of new treatment options." European regulators reviewed Novartis' marketing application under an accelerated assessment granted in January. 

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Novartis did not provide information regarding what it intends to charge for Kymriah, whose price was set at $475 000 for a one-time single administration in the US, where it was approved last year to treat ALL in certain children and young adults, marking the first authorisation of a gene therapy in the country. The label was later expanded to include certain patients with r/r large B-cell lymphoma. The company indicated that it will continue discussions with officials "on a fair, value-based pricing approach that is sustainable for national healthcare systems." 

Novartis stated it expects to initially launch Kymriah in the paediatric ALL indication, "as we continue to ramp up capacity." The company noted that "timing for Kymriah availability in each country will depend on multiple factors, including the onboarding of qualified treatment centres for the appropriate indications, as well as the completion of national reimbursement procedures."

Separately, Novartis announced plans to expand a production site in Aargau, Switzerland, for cell and gene therapies, creating 260 new jobs, with the potential to raise that number to 450 by 2021. In May of last year, the company said it would eliminate up to 500 jobs in the Basel area, affecting staff in the traditional production, coordination and development fields, but that it would add another 350 high-tech positions mainly in the development and production of innovative biotechnology medicines. The latest move follows Novartis' recent decision to begin producing cell and gene therapies in France via an agreement with CELLforCURE.


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