Regulatory Affairs

FDA expands approval of Novartis’ Kymriah CAR-T therapy

PBR Staff Writer Published 02 May 2018

Novartis has secured a second approval for Kymriah (tisagenlecleucel) in the US for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma who had more than two lines of systemic therapy.

The new indication approved by the US Food and Drug Administration (FDA) covers diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma and DLBCL.

The immunocellular therapy has not been indicated for the treatment of primary central nervous system lymphoma, said Novartis.

Kymriah’s first approval from the FDA came in August 2017 for the treatment of children and young adults with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

A chimeric antigen receptor T cell (CAR-T) therapy, Kymriah is a one-time treatment that uses a patient's own T cells to treat cancer. Developed in collaboration with the University of Pennsylvania, Kymriah is manufactured individually for each patient.

Kymriah’s latest approval by the FDA for treating adult patients with r/r DLBCL has been driven by the results of a phase II trial called JULIET.

In this global trial, Kymriah registered an overall response rate (ORR) of 50%, with 32% of patients achieving a complete response (CR) while 18% of them achieved a partial response (PR) in 68 patients, who were evaluated for efficacy.

Novartis Oncology CEO Liz Barrett said: "Today's FDA approval of Kymriah provides another opportunity for Novartis to build on its leadership in CAR-T development, delivering a potentially transformative therapy with durable and sustained response rates and a well-characterized safety profile to help patients in dire need of new treatment options.

"We look forward to leveraging all of our learnings and new capabilities from the initial launch of Kymriah in pediatric and young adult B-cell ALL for this larger group of patients."

Earlier, this week, Novartis got an approval from FDA for the Tafinlar (dabrafenib) and Mekinist (trametinib) combination for the adjuvant treatment of melanoma in patients who are positive to BRAF V600E or V600K mutations, as detected by an FDA-approved test, and involvement of lymph nodes, after complete resection.


Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis AG.