FDA accepts a Biologics License Application for obe-cel in patients with R/R B-ALL

On January 22, 2024, the U.S. Food and Drug Administration (FDA) accepted a Biologics License Application for obecabtagene autoleucel (obe-cel), a novel autologous CD19 CAR T-cell therapy designed with a fast off-rate CD19 binding domain to improve persistence and reduce toxicities, for the treatment of adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia. This decision was based on data from the pivotal phase II FELIX study (NCT04404660).^1,2

The ALL hub previously reported the efficacy and safety outcomes from the FELIX study. Updated data was presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition. A total of 127 patients were infused with obe-cel. By the data cut-off on September 13, 2023,

• 74% of patients with morphological disease (n = 98) achieved a complete remission/complete response with incomplete count recovery rate;
• 95% of responders were measurable residual disease-negative;
• high complete response/complete response with incomplete count recovery rates were seen across all subgroups, including those with high-burden and extramedullary disease;
• the 12-month event-free survival rate was 50%; and
• obe-cel demonstrated long-term persistence and a favorable safety profile, with low rates of Grade ≥3 cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome reported.²

For this indication, obe-cel previously received Orphan Drug Designation and Regenerative Medicine Advanced Therapy designation by the FDA, as well as Orphan Medical Product Designation and Priority Medicines designation by the European Medicines Agency (EMA).¹