FDA approves Kymriah CAR-T cell therapy for adult patients with relapsed or refractory follicular lymphoma .- Novartis

In accordance with the Accelerated Approval Program, continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Kymriah is now FDA approved in three indications and remains the only CAR-T cell therapy approved in both adult and pediatric settings.

The approval is based on data from the Phase II ELARA trial, a single-arm, open-label trial, in which 90 patients were evaluated for efficacy with a median follow-up of approximately 17 months. Eighty-six percent of patients treated with Kymriah achieved a response including 68% who experienced a complete response. Prolonged durable response to treatment was demonstrated with an estimated 85% of patients who achieved a complete response still in response 12 months after initial response1. Kymriah was 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 Follicular Lymphoma International Prognostic Index (FLIPI) scores.

For the 97 patients evaluable for safety at 21 months of median follow-up, the safety profile of Kymriah was remarkable. Fifty-three percent of patients experienced any-grade cytokine release syndrome (CRS), as defined by the Lee scale, and there were no reported cases of high-grade (grade 3 or higher) CRS. Forty-three percent of patients experienced any-grade neurologic events; grade 3 or higher neurologic events were seen in only 6% of patients. Eighteen percent of patients (17 of 97 patients) were infused in an outpatient setting. .