Updated: FDA Approves Aucatzyl for Adults with Aggressive Leukemia

The FDA has approved Aucatzyl (obecabtagene autoleucel or obe-cel) to treat adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).

Acute lymphoblastic leukemia is a fast-growing cancer in which B-cell lymphoblasts are found in the bone marrow and blood. It accounts for less than half of 1% of all cancers in the United States. The American Cancer Society estimates that in 2024 there will be about 6,550 new cases of ALL and about 1,330 deaths.

Approximately 8,400 new cases of adult ALL are diagnosed every year in the United States and European Union, with around 3,000 patients in the relapsed refractory setting. Survival rates remain very poor in adult patients with r/r ALL, with median overall survival of eight months.

Elias Jabbour, M.D.

“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,” Elias Jabbour, M.D., U.S. lead investigator and professor of Leukemia, ALL Section Chief, at The University of Texas MD Anderson Cancer Center, Houston, said in a news release.

Developed by Autolus Therapeutics, Aucatzyl is a B-lymphocyte antigen CD19 chimeric antigen receptor (CAR) T cell therapy. It was designed to minimize excessive activation of the programmed T cells. The wholesale acquisition cost is $525,000 annually. Company officials said pricing reflects clinical evidence, differentiated safety profile, and economic value of Aucatzyl.

The company said in an investor call that 30 centers that provide care for about 60% of r/r B-ALL target population are prepared to begin treating patients, with 30 additional centers to follow by end 2025.

The approval of Aucatzyl was based on data from the FELIX clinical trial. In the 65 patients evaluated for efficacy, 63% achieved overall complete remission (OCR), which includes 51% of patients with complete remission at any time and 12% patients with complete remission with incomplete hematologic recover at any time. The major efficacy outcome was complete remission within 3 months, which was achieved in 42% patients, and the median duration of remission was 14.1 months.

Aucatzyl showed low levels of cytokine release syndrome (CRS), with 3% grade 3 events, and no grade 4 or 5 events. The label includes a boxed warning about the risk of cytokine release syndrome, neurologic toxicities, and secondary hematological malignancies.

According to company officials, Aucatzyl is the first CAR T-cell therapy approved by the FDA with no requirement for a Risk Evaluation and Mitigation Strategy (REMS) program.

This story was updated with information from a company call with investors.