FDA approves Autolus CAR-T therapy for acute lymphoblastic leukemia

The U.S. FDA has approved Autolus Therapeutics' obecabtagene autoleucel, branded Aucatzyl, a CD19-directed genetically modified autologous T-cell immunotherapy for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).

The milestone approval, which marks Autolus' first commercial product, offers new hope for patients with this aggressive cancer once they relapse.

Aucatzyl is to be administered as a split dose infusion on day 1 and day 10 (±2 days) based on bone marrow blast assessment and preceded by fludarabine and cyclophosphamide lymphodepleting chemotherapy. 

The approval is based on the FELIX clinical trial, where 42% of patients achieved complete remission within three months, and the median duration of remission was 14.1 months. According to Autolus, Aucatzyl showed low levels of cytokine release syndrome (CRS) — a common and serious side effect of CAR-T therapy — with 3% grade 3 events, and no grade 4 or 5 events. 

Like other CAR-T products, Aucatzyl comes with a boxed warning for CRS, neurologic toxicities, and secondary hematological malignancies. Aucatzyl does not have a REMS requirement, reducing ongoing reporting obligations.  The move is in line with the agency's efforts to reduce the burden on the health care delivery system related to CAR-T therapies. Back in June, the FDA modified the REMS for several approved CAR-T therapies, removing requirements for educational and training materials as well as the requirement to report adverse events suggestive of CRS or neurological toxicities.

According to Autolus, marketing authorization applications for obe-cel in adult r/r ALL are being reviewed by the regulators in both the EU and the UK, with a submission to the EMA accepted in March 2024, and a submission accepted by the UK MHRA in August 2024.