NEW YORK – The US Food and Drug Administration on Friday approved Autolus Therapeutics' CD19-directed autologous CAR T-cell therapy Aucatzyl (obecabtagene autoleucel) as a treatment for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (B-ALL).
The approval, the first for Aucatzyl, was based on results from the single-arm Phase I/II FELIX trial, which included 65 evaluable patients with relapsed or refractory CD19-positive B-ALL who either had relapsed following a remission lasting 12 months or less, had relapsed or refractory ALL after two or more prior lines of systemic therapy, or had relapsed or refractory disease three or more months after allogeneic stem cell transplantation.
Autolus was primarily interested in the number of patients who achieved a complete remission within three months after receiving its CAR T-cell therapy. In the trial, 27 patients, or 42 percent, achieved a complete remission within three months on Aucatzyl. The median duration of these remissions was 14.1 months.
Among the 65 evaluable patients, about half, 51 percent, had a complete remission at any time, and 12 percent experienced a complete remission with incomplete hematologic recovery at any time. In total, 63 percent of patients in the trial achieved a complete remission at any point.
Aucatzyl's FDA-approved label includes a boxed warning for cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, and T-cell malignancies. In the trial, cytokine release syndrome occurred in 75 percent of patients with 3 percent having a grade 3 event, and neurologic toxicities occurred in 64 percent with 12 percent experiencing grade 3 or higher neurologic toxicities.
Autolus noted in its announcement that Aucatzyl is the first CAR T-cell therapy approved in the US that does not have a required Risk Evaluation Mitigation Strategy (REMS) program.
"Based on the experience in the FELIX trial, Aucatzyl is highly active and can be well managed, offering an attractive risk-benefit profile for B-ALL patients," Claire Roddie, lead investigator of the FELIX study and associate professor of hematology at the University College London Cancer Institute, said in a statement. "In the FELIX trial, Aucatzyl has shown long-term persistence and deep responses, which we believe are critical for long-term remissions in B-ALL."
Earlier this year, Autolus submitted a marketing authorization application for Aucatzyl to the European Medicines Agency seeking approval in this same indication.