On April 25, 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 UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation for AUCATZYL (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL) (Press release, Autolus, APR 25, 2025, View Source [SID1234652154]).
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"Having treated a number of patients with AUCATZYL as part of the FELIX clinical trial, I am delighted that we have moved closer to eligible relapsed/refractory B-ALL patients being able to access AUCATZYL," said 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. "We now look forward to NICE completing its assessment of the medicine to potentially make it an option for eligible patients on the NHS."
"AUCATZYL was designed to address an unmet need for eligible adult r/r B-ALL patients and it is satisfying that is has been licensed in the country where it was created," said Dr. Martin Pule, Chief Scientific Officer and Founder of Autolus.
"Continuing our momentum, this MHRA license is a significant milestone for Autolus as a company. With our scientific expertise, operations and manufacturing based in the UK, this is an important achievement for our company," said Dr. Christian Itin, Chief Executive Officer of Autolus. "We want to thank all the patients and investigators at the UK trial centres for their contributions towards this license, as well as the foundational work by our partners at UCL and our internal team."
Obecabtagene autoleucel 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.
The MHRA authorisation of AUCATZYL 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 of which were published in the New England Journal of Medicine in November 20242. Of the 153 r/r B-ALL patients enrolled in the FELIX study, 127 (83.0%) received at least one obecabtagene autoleucel infusion and were evaluable. 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%).
For further information regarding obecabtagene autoleucel, the Summary of Product Characteristics (SPC), including a full list of side effects and adverse reactions, is available here.
Autolus submitted obecabtagene autoleucel for appraisal by the National Institute for Health and Care Excellence (NICE)3 in Q4 2024 and is working with NICE and the NHS to potentially achieve access for eligible patients in England. NICE provides guidance to the NHS in England on the clinical and cost-effectiveness of medicines, treatments, and technologies based on a rigorous process of evidence review and consultation with professionals and patients.
ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system and other organs. In the UK there are approximately 7654 new cases of ALL diagnosed every year. In frontline treatment for adult B-ALL, up to 50% of patients will ultimately relapse5. Survival rates remain very poor in adult patients with r/r ALL, with median overall survival of eight months with conventional treatments6, and the standard-of-care treatment can trigger severe toxicities.