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The FDA approved obecabtagene autoleucel (obe-cel; Aucatzyl) for treating relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in adults, the on Friday.

Of note, unlike other CAR T-cell therapies, the CD19-directed genetically modified autologous T-cell immunotherapy will have no requirement for a Risk Evaluation and Mitigation Strategy (REMS) program.

Approval was supported by the single-arm, multicenter , which enrolled patients with relapsed or refractory CD19-positive B-cell ALL.

Among the 65 patients evaluable for efficacy, a single infusion of obe-cel yielded a complete remission (CR) rate of 42% (95% CI 29-54) at 3 months. Of these responders, the median duration of CR reached 14.1 months. Overall, 63% of participants achieved a CR during the study, including 12% with CR and incomplete hematologic recovery.

"Aucatzyl is highly active and can be well managed, offering an attractive risk-benefit profile for B-ALL patients," investigator Claire Roddie, MD, PhD, of the University College London Cancer Institute, said in a from drugmaker Autolus Therapeutics. "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."

Patients enrolled in the phase Ib/II trial had to have relapsed following a prior remission of a year or less, have relapsed or refractory ALL after at least two previous lines of therapy, or have disease that relapsed or was refractory 3 or more months after allogeneic stem-cell transplantation.

"Adult ALL is an extremely aggressive cancer, and there is a high unmet medical need that exists in the treatment of patients with this disease once they relapse, where historically they suffer from poor outcomes," investigator Elias Jabbour, MD, of the University of Texas MD Anderson Cancer Center in Houston, said in a statement.

Like other CAR T-cell therapies, the has a boxed warning over risks of cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and secondary T-cell malignancies. In the FELIX trial, CRS occurred in 75% of patients (with 3% grade 3 in severity), while ICANS occurred in 24% (with 7% grade ≥3). Overall, neurologic toxicities occurred in 64% (12% grade ≥3).

Other common non-laboratory adverse events, occurring in 20% or more of participants, included infections and bacterial infectious disorders, musculoskeletal pain, fever, nausea, diarrhea, febrile neutropenia, hypotension, pain, fatigue, headache, encephalopathy, and hemorrhage.

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