On July 21, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies and candidates, reported that the European Commission (EC) has granted marketing authorization for AUCATZYL (obecabtagene autoleucel or "obe-cel") for the treatment of adult patients, 26+, with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL) (Press release, Autolus, JUL 21, 2025, View Source [SID1234654458]).
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The EC approval was based on the results of the FELIX study, an open-label, multi centre, single arm study in adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia. The results were published in the New England Journal of Medicine in November 20241. In the pivotal cohort of patients, (cohort IIA (n=94)), the Complete Response/Complete Response with Incomplete Haematological Recovery (CR/CRi) for patients who received at least one infusion of obecabtagene autoleucel was 76.6%. Median response duration for all infused patients was 21.2 months. Median event-free survival (EFS) was 11.9 months and the estimated 6- and 12-month event-free survival rates were 65.4% and 49.5%, respectively.
The most common non-laboratory Grade 3 or higher adverse reactions were infections-pathogen unspecified (32%), febrile neutropenia (24%) and bacterial infectious disorders (11%). Cytokine release syndrome developed in 87 of the 127 patients (68.5%), with events of grade 3 or higher in three patients (2.4%). Immune effector cell-associated neurotoxicity syndrome developed in 29 of the 127 patients (22.8%), with grade 3 or higher occurring in nine patients (7%).
"We believe AUCATZYL represents an important new treatment option for physicians treating adult r/r B-ALL patients. With the EU marketing authorization, we are now evaluating market entry opportunities in EU countries," said Dr. Christian Itin, Chief Executive Officer of Autolus.
Obe-cel is an autologous CD19 CAR T cell therapy with a proprietary CD19 CAR, invented by a team led by Dr. Martin Pule, at University College London, along with collaborators at Great Ormond Street Hospital and University College London Hospital. The CAR is designed to have a fast "off-rate" which mimics physiological T-cell receptor interactions2.
ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system and other organs. In Europe, there are approximately 6,0002 new cases of ALL diagnosed every year. In frontline treatment for adult B-ALL, up to 50% of patients will ultimately relapse3. Survival rates remain very poor in adult patients with r/r ALL, with median overall survival of eight months with conventional treatments4, and the standard-of-care treatment can trigger severe toxicities5.
The EC approval applies to all 27 European Union Member States, Iceland, Norway and Liechtenstein. AUCATZYL is currently approved by the U.S. Food and Drug Administration (FDA) and authorized by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA).