FDA Approves New CAR T-Cell Therapy for Relapsed, Refractory Follicular Lymphoma
Tisagenlecleucel was shown to be effective in high-risk patients, including those who were heavily pretreated or had refractory disease.
Officials with the FDA have approved tisagenlecleucel (Kymriah; Novartis) for the treatment of adults with relapsed or refractory follicular lymphoma after 2 or more lines of systemic therapy.
The approval is part of the FDA’s Accelerated Approval Program, so its continued approval may be contingent on verification and description of clinical benefit in confirmatory trials. This marks the third indication for tisagenlecleucel, which remains the only chimeric antigen receptor T-cell therapy approved in both adult and pediatric patients.
“We are proud of today’s FDA approval of a third indication for Kymriah,” said Victor Bulto, MBA, president of Novartis Innovative Medicines US, in a press release. “We hope this treatment option that has the potential for long-lasting results may help break the unrelenting cycle of treatment for patients with follicular lymphoma.”
The approval is based on data from the phase 2 ELARA trial, in which 90 patients were evaluated for efficacy with a median follow-up of approximately 17 months. According to clinical trial results, 68% of patients receiving tisagenlecleucel in the ELARA trial experienced a complete response, with an 86% overall response rate. Sustained clinical benefit from the treatment was also demonstrated, with 85% of patients who achieved a complete response remaining in response at 12 months.
Tisagenlecleucel was also shown to be effective in high-risk patients, including those who were heavily pretreated or had refractory disease, POD24, bulky disease, or those with high scores on the Follicular Lymphoma International Prognostic Index.
Of the 97 patients evaluable for safety at 21 months of median follow-up, researchers found a “remarkable” safety profile, according to the study. Fifty-three percent of patients experienced any-grade cytokine release syndrome (CRS) and there were no reported cases of high-grade CRS. Furthermore, 43% of patients experienced any-grade neurologic events and grade 3 or higher neurologic events were seen in just 6% of patients. Finally, 18% of patients (17 of 97) were infused in an outpatient setting.
“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, institutional principal investigator on the ELARA trial, in the press release. “This new, effective option for patients with follicular lymphoma may offer long-term benefit.”
Researchers noted that although follicular lymphoma is typically an indolent form of cancer, patients may be exposed to a median of 4 lines of treatment, with an upper range of 13 lines. Importantly, despite the availability of multiple systemic therapies, the efficacy of these regimens decreases sharply in later lines.
“The approval of Kymriah offers patients with relapsed or refractory follicular lymphoma a new treatment option and new hope for improving patient outcomes,” said Meghan Gutierrez, CEO at the Lymphoma Research Foundation, in the press release. “Having this single infusion treatment option helps to transform the way health care providers approach this type of blood cancer and we commend those who have contributed to the acceleration of scientific research for the benefit of patients.”
FDA approves Novartis Kymriah CAR T-cell Therapy for Adult Patients With Relapsed or Refractory Follicular Lymphoma. News release. Novartis; May 28, 2022. Accessed May 31, 2022. https://www.novartis.com/news/media-releases/fda-approves-novartis-kymriah-car-t-cell-therapy-adult-patients-relapsed-or-refractory-follicular-lymphoma