The Food and Drug Administration (FDA) has granted accelerated approval to Kymriah® (tisagenlecleucel), a CD19-directed chimeric antigen receptor (CAR) T-cell therapy, 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 principal investigator Stephen J. Schuster, MD, of the University of Pennsylvania’s Abramson Cancer Center. “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
This article originally appeared on MPR