FDA Approves CAR T-Cell Treatment Kymriah for Adult B-Cell Lymphoma

The FDA has approved Novartis' chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (Kymriah) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma, the most common form of non-Hodgkin lymphoma.

The indication includes diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma after 2 or more lines of systemic therapy. For patients who do not respond to initial therapy, limited treatment options are available that provide durable response, and median life expectancy is approximately 6 months.

The approval marks tisagenlecleucel's second indication. In August 2017, the therapy became the first CAR T-cell therapy approved for the treatment of B-cell precursor acute lymphoblastic leukemia in certain pediatric and young adult patients.

“The goal of Kymriah is to provide physicians with a therapy that has demonstrated durable response rates in relapsed or refractory DLBCL patients, a patient population that has endured multiple rounds of chemotherapy with many having experienced unsuccessful stem cell transplants,” said Stephen J. Schuster, MD, Robert and Margarita Louis-Dreyfus professor in chronic lymphocytic leukemia and lymphoma clinical care and research in Penn’s Perelman School of Medicine and director of the Lymphoma Program at the Abramson Cancer Center. “With this approval, physicians now have a meaningful therapeutic options that can achieve and maintain a sustained response without stem cell transplant along with a consistent safety profile.”

The approval follows results of the pivotal phase 2 JULIET study, which enrolled patients with r/r DLBCL from 27 sites in 10 countries. Patients showed progression of disease after receiving at least 2 lines of chemotherapy and were ineligible or failed autologous stem cell transplant. Tisagenlecleucel showed an overall response rate of 50% (95% CI, 38%-62%), with 32% of patients achieving a complete response and 18% of patients achieving a partial response. The median duration of response had not been reached.

The therapy will compete with Gilead Science’s CAR T-cell therapy axicabtagene ciloleucel (Yescarta), which was approved in October 2017 for adult patients with B-cell lymphoma.