Autolus Therapeutics plc (Nasdaq: AUTL) today announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for AUCATZYL™ (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).
The approval marks a significant advancement in the treatment landscape for adult ALL, an aggressive cancer with historically poor outcomes in relapsed settings. According to Dr. Elias Jabbour, U.S. lead investigator of the FELIX study, this approval “brings new hope for adult patients with relapsed/refractory B-ALL.”
Efficacy and Safety Data from the FELIX Trial
The FDA's decision was supported by data from the FELIX clinical trial, which evaluated the efficacy and safety of obe-cel in adult patients with r/r B-ALL. In the morphological disease cohort, 94 patients received at least one infusion of AUCATZYL, with 65 patients having >5% blasts in the bone marrow after screening and prior to lymphodepletion therapy. Among the efficacy-evaluable patients (n=65), 63% achieved overall complete remission (OCR), including 51% with complete remission (CR) and 12% with complete remission with incomplete hematologic recovery (CRi).
The major efficacy outcome of complete remission within 3 months was achieved in 42% of patients, and the median duration of remission (DOR) was 14.1 months. The safety profile of AUCATZYL was notable for low rates of severe Cytokine Release Syndrome (CRS), with only 3% Grade 3 events and no Grade 4 or 5 events. Grade ≥ 3 Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) was reported in 7% of patients. Importantly, the FDA did not require a REMS program for AUCATZYL.
Dr. Claire Roddie, lead investigator of the FELIX study, noted that AUCATZYL is “highly active and can be well managed, offering an attractive risk benefit profile for B-ALL patients.” She added, “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.”
Manufacturing and Commercialization
AUCATZYL will be manufactured at Autolus’ dedicated commercial manufacturing site, the Nucleus, in Stevenage, UK. The facility has received authorization from both the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and the FDA. Autolus will partner with Cardinal Health for commercial distribution in the U.S. The company will now work with treatment centers to onboard and initiate patient scheduling, with commercial availability expected soon.
About B-ALL
Acute lymphoblastic leukemia (ALL) is an aggressive blood cancer that affects the lymph nodes, spleen, liver, central nervous system, and other organs. Approximately 8,400 new cases of adult ALL are diagnosed annually in the US and EU, with about 3,000 patients in the relapsed/refractory setting. Survival rates for adult patients with r/r ALL remain poor, with a median overall survival of eight months.
Ongoing Regulatory Reviews
Marketing authorization applications (MAAs) for obe-cel in adult r/r ALL are currently under review by regulators in the EU and the UK, with submissions accepted by the European Medicines Agency (EMA) in March 2024 and by the UK MHRA in August 2024.
