The US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah (tisagenlecleucel) by Novartis for the treatment of adult patients with relapsed or refractory (r/r) Follicular Lymphoma (FL) after two or more lines of systemic therapy. In accordance with the Accelerated Approval Programme, 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 paediatric settings, Novartis recently announced via a statement.
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 per cent, who experienced a complete response, according to the statement.
Prolonged durable response to treatment was demonstrated with an estimated 85 per cent of patients who achieved a complete response still in 12 months after initial response. Kymriah was shown to be effective in high-risk patients, including those who were heavily pre-treated or had refractory disease, POD24, bulky disease or those with high Follicular Lymphoma International Prognostic Index (FLIPI) scores, the statement said.
It added that 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-III or higher) CRS. Forty-three per cent of patients experienced any-grade neurologic events; grade-III or higher neurologic events were seen in only six per cent of patients. Eighteen per cent of patients (17 of 97 patients) were infused in an outpatient setting.
In early May 2022, the European Commission had approved Kymriah for the treatment of adult patients with r/r FL after two or more lines of systemic therapy, the third indication for which Kymriah is available to patients in the European Union (EU), the statement concluded.