The Food and Drug Administration (FDA) has granted accelerated approval to Kymriah® (tisagenlecleucel), a CD19-directed genetically modified autologous T-cell immunotherapy, for the treatment of adults with relapsed or refractory follicular lymphoma after 2 or more lines of systemic therapy.
The approval was based on data from the multicenter, single-arm, open-label phase 2 ELARA trial (ClinicalTrials.gov Identifier: NCT03568461). The efficacy and safety of Kymriah was evaluated in adults with follicular lymphoma who were refractory to or relapsed within 6 months after completion of 2 or more lines of systemic therapy (including an anti-CD20 antibody and an alkylating agent), relapsed during or within 6 months after completion of an anti-CD20 antibody maintenance therapy following at least 2 lines of therapy, or relapsed after autologous hematopoietic stem cell transplant.
Results showed an overall response rate of 86% (77/90), with 68% (61/90) of patients experiencing a complete response; the median time to first response was 2.9 months (range, 0.6 to 6.0 months). Among patients who achieved a complete response, 85% were still in response at 12 months.
“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,” said Stephen J. Schuster, MD, 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, institutional principal investigator on the trial. “This new, effective option for patients with follicular lymphoma may offer long-term benefit.”
Continued approval of Kymriah for relapsed or refractory follicular lymphoma may be contingent upon verification and description of clinical benefit in a confirmatory trial. The accelerated approval was based on response rate and duration of response.
Kymriah carries a Boxed Warning regarding cytokine release syndrome and neurological toxicities. It is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Kymriah REMS.
- FDA approves Novartis Kymriah® CAR-T cell therapy for adult patients with relapsed or refractory follicular lymphoma. News release. Novartis US. Accessed May 31, 2022. https://www.prnewswire.com/news-releases/fda-approves-novartis-kymriah-car-t-cell-therapy-for-adult-patients-with-relapsed-or-refractory-follicular-lymphoma-301556984.html
- Kymriah. Package insert. Novartis US; 2022. Accessed May 31, 2022. https://www.novartis.us/sites/www.novartis.us/files/kymriah.pdf