Autolus Therapeutics plc (NASDAQ: AUTL) announced that the U.S. Food and Drug Administration (FDA) has approved AUCATZYL® (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). This approval marks a significant advancement in the treatment landscape for this aggressive cancer, addressing a high unmet medical need.
The FDA's decision was based on the results of the pivotal FELIX clinical trial, which evaluated the efficacy and safety of obe-cel in adult patients with r/r B-ALL. The study demonstrated a clinically meaningful benefit, offering new hope for patients facing limited treatment options.
Efficacy and Safety Data from the FELIX Trial
The FELIX trial included adult patients with r/r B-ALL who had received prior therapies and experienced relapse or were refractory to existing treatments. Key findings from the efficacy-evaluable patients (n=65) showed:
- An overall complete remission (OCR) rate of 63%, comprising 51% with complete remission (CR) and 12% with complete remission with incomplete hematologic recovery (CRi).
- Complete remission within 3 months was achieved in 42% of patients.
- The median duration of remission (DOR) was 14.1 months.
In terms of safety, AUCATZYL demonstrated manageable toxicity. Cytokine Release Syndrome (CRS) occurred at low levels, with only 3% of patients experiencing Grade 3 events and no Grade 4 or 5 events. Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) of Grade ≥ 3 was reported in 7% of patients. Importantly, the FDA did not require a REMS program for AUCATZYL, highlighting its manageable safety profile.
Clinical Perspective
"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," said Elias Jabbour, MD, U.S. lead investigator of the FELIX study and professor of Leukemia, ALL Section Chief, at The University of Texas MD Anderson Cancer Center, Houston, Texas. "This milestone approval, based on the demonstrated clinical benefit of AUCATZYL, brings new hope for adult patients with relapsed/refractory B-ALL."
Dr. Claire Roddie, MD, PhD, FRCPath, Lead investigator of the FELIX study and Associate Professor of Haematology at the University College London (UCL) Cancer Institute, added, "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. 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 the necessary authorizations and certifications from regulatory agencies, including the MHRA in the UK. Autolus has partnered with Cardinal Health for commercial distribution in the U.S. The company will now work with treatment centers to onboard them and initiate patient scheduling, aiming to make AUCATZYL commercially available in the U.S. promptly.
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. The survival rates for adult patients with r/r ALL remain poor, with a median overall survival of approximately eight months.
Ongoing Regulatory Reviews
Marketing authorization applications (MAAs) for obe-cel in adult r/r ALL are currently under review by regulatory bodies in both the EU and the UK. The European Medicines Agency (EMA) accepted the submission in March 2024, and the UK MHRA accepted the submission in August 2024.
