Novartis announced that the FDA authorised Kymriah (tisagenlecleucel) for certain patients with relapsed or refractory (r/r) large B-cell lymphoma, marking the second approval in the US for the CAR-T therapy. Specifically, Kymriah is cleared for the treatment of adults with diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma and DLBCL arising from follicular lymphoma, after two or more lines of systemic therapy.
In 2017, the immunocellular therapy gained FDA approval for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory or in second or later relapse. Kymriah, which is a one-time treatment manufactured individually for each patient using the patient's own T cells, was developed in collaboration with the University of Pennsylvania.
Novartis noted that approval of Kymriah in adults with r/r DLBCL is based on a submission including data from the pivotal Phase II JULIET study, with results showing that the therapy was associated with an overall response rate of 50 percent in 68 patients evaluated for efficacy. Findings demonstrated that 32 percent of patients achieved a complete response and 18 percent had a partial response.
In January, the European Medicines Agency granted accelerated assessment to an application for Kymriah for the treatment of children and young adults with r/r B-cell ALL and for adults with r/r DLBCL who are ineligible for autologous stem cell transplant.
Last month, Novartis reported that first-quarter sales of Kymriah reached $12 million. The company priced Kymriah at $475 000 for patients with B-cell precursor ALL, although the cost is only recovered if patients respond within 30 days of treatment, while the treatment is priced at $373 000 in the DLBCL indication. Pascal Touchon, head of cell and gene oncology at Novartis, noted that clinical studies have shown that a significant number of patients with DLBCL may not respond to Kymriah until several months after treatment, making it difficult to measure outcomes within a defined period of time.
In October last year, the FDA granted approval to Gilead Sciences' Kite unit for its CAR-T therapy Yescarta (axicabtagene ciloleucel) for certain patients with large B-cell lymphoma, with the company pricing the immunotherapy at $373 000.
For related analysis, see ViewPoints: A CAR-T showdown, and ViewPoints: CAR-Ts make baby steps on securing reimbursement.
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