The approval includes adults with R/R DLBCL after two or more lines of prior systemic therapy, high-grade B-cell lymphoma, and DLBCL arising from FL.
The US Food and Drug Administration (FDA) has expanded the approval of the chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (Kymriah; Novartis) to include treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of prior systemic therapy, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (FL). These indications are in addition to its prior indication for children and young adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
“This is an exciting event-seeing this lifesaving therapy become available widely to a large patient population with an unmet medical need,” Stephen J. Schuster, MD, director of the Lymphoma Program at the University of Pennsylvania’s Abramson Cancer Center said in a press release.
The approval was based on the results of two studies looking at CAR T-cell therapy in DLBCL. The first, published in The New England Journal of Medicine, included 28 adults with relapsed or refractory lymphoma treated with a single infusion of the CD19-directed CAR therapy. Sixty-four percent of patients had a response to treatment. Complete response occurred in 6 of 14 patients with DLBCL and 10 of 14 patients with FL. With a median follow-up of 28.6 months, response was maintained in 86% of patients with DLBCL and 89% of patients with FL.
Results from the second trial, JULIET, were presented at the 59th American Society of Hematology annual Meeting in December. Among the 81 patients infused with CAR therapy in this trial, the overall response rate (ORR) was 53%, with 40% of patients achieving complete response and 14% achieving partial response. At 6 months, the ORR was 37%, with a complete response rate of 30%. By 6 months, a median duration of response was not yet reached.
In a press release about the approval, Novartis said it is committed to ensuring that eligible patients have access to tisagenlecleucel. The company continues to collaborate with the Centers for Medicare and Medicaid Services (CMS) on the creation of an appropriate value-based pricing approach. According to previous announcements, therapy could cost about $475,000 per treatment for pediatric leukemia and $373,000 for the new lymphoma indication.