FDA Approves Kymriah for Adults with Follicular Lymphoma

Kymriah is now approved in three indications and is the only CAR-T cell therapy approved in both adult and pediatric settings.

The FDA has granted accelerated approval for Novartis’ Kymriah (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. Kymriah is now FDA approved in three indications and is the only chimeric antigen receptor (CAR)-T cell therapy approved in both adult and pediatric indications.

The approval is based on data from the phase 2 ELARA trial, a single-arm trial, in which 90 patients were evaluated for efficacy with a median follow-up of about 17 months. Eighty-six percent of patients treated with Kymriah achieved a response, including 68% who experienced a complete response.

In an extended study, 97 patients were evaluated for safety at 21 months of median follow-up. Of these, 53% experienced cytokine release syndrome (CRS), an inflammatory response characterized by fever and multiple organ dysfunction. It is caused by a large, rapid release of cytokines into the blood from immune cells.

“Patients with follicular lymphoma who relapse or don’t respond to treatment have a poor prognosis and may face a series of treatment options without a meaningful, lasting response,” Principal Investigator Stephen J. Schuster, M.D., said in a press release. He is the Robert and Margarita Louis-Dreyfus Professor in Chronic Lymphocytic Leukemia and Lymphoma in the Division of Hematology Oncology and Director, Lymphoma Program and Translational Research at the University of Pennsylvania’s Abramson Cancer Center.

Follicular lymphoma is the second most common form of non-Hodgkin lymphoma, representing about 22% of non-Hodgkin lymphoma cases. It is often an unrelenting malignancy with a relapsing and remitting pattern, and patients may be exposed to a median of five lines of prior treatment. The efficacy of these regimens drops off rapidly in later lines of treatment.

Kymriah is also approved to treat children and young adults with B-cell acute lymphocytic leukemia and adults with relapsed or refractory large B-cell lymphoma.