The Medicines and Healthcare products Regulatory Agency (MHRA) has today (25 April 2025) granted a conditional marketing authorisation for the medicine obecabtagene autoleucel (Aucatzyl), a chimeric antigen receptor (CAR) T-cell therapy, to treat adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).
B-cell precursor acute lymphoblastic leukaemia is a type of blood cancer that affects the white blood cells, specifically the B-lymphocytes. In the condition, the bone marrow produces a large number of abnormal, immature B-lymphocytes, often known as blast cells which grow and divide quickly.
For relapsed patients with ALL, it means their leukaemia has returned after a period of improvement or remission following initial treatment, whereas for refractory patients, it means their leukaemia did not respond sufficiently to initial treatment.
Acute lymphoblastic leukaemia is rare, affecting less than 5 in 10,000 people in the UK.
Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said
“Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.
“We are committed to making innovative treatment options, like CAR T-cell therapy, available to patients as quickly as possible, ensuring our approval is underpinned by robust evidence of efficacy alongside the highest standards of safety. We are assured that the appropriate regulatory standards for the approval of this medicine have been met.
“As with all products, we will keep the safety of obecabtagene autoleucel under close review.”
The medicine is given as an intravenous (IV) infusion in a clinical setting by a physician with experience in the treatment of haematological malignancies and trained for administration and management of patients treated with this medicine.
Obecabtagene autoleucel is a type of immunotherapy call CAR-T therapy that works by taking a patient’s T cells, a type of white blood cell, and putting them through a process that transforms them into CAR T cells that are able to target the CD19 protein. When put back into the body, these modified cells can recognise and destroy the cancer cells.
This conditional approval is supported by evidence from the FELIX study, an ongoing open-label, single-arm study which enrolled 153 adult patients with relapsed or refractory B cell acute lymphoblastic leukaemia. Of those patients, 94 were administered at least one infusion of obecabtagene autoleucel.
Prognosis for patients with relapsed or refractory B cell acute lymphoblastic leukaemia is typically poor with short overall survival rates reported. In this study, 52 of the 94 patients given the medicine showed complete remission of the disease with an 81% probability of overall survival at 12 months.
The most common side effects of the medicine (which may affect more than 1 in 10 people) include nausea, headache, abnormal brain function, dizziness, fever and low blood pressure. Patients are advised to refer to the Patient Information Leaflet for a full list of side effects.
As with any medicine, the MHRA will keep the safety and effectiveness of obecabtagene autoleucel under close review.
Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme, either through the website (https//yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.
Notes to editors
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The new conditional marketing authorisation was granted on 25 April 2025 to Autolus Therapeutics.
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More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
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Obecabtagene autoleucel (Aucatzyl) has been conditionally approved through the national assessment procedure.
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A conditional marketing authorisation (CMA) is an early temporary licence in which we may accept less completed clinical studies than would be necessary to issue a full marketing authorisation, provided the manufacturer clearly indicates when complete clinical data will be available. However, CMA post-approval conditions are determined on a case-by-case basis, and don’t have to be limited to providing further clinical data. A CMA lasts for one year and can be renewed annually.
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For more information about acute lymphoblastic leukaemia (ALL), visit https//www.nhs.uk/conditions/acute-lymphoblastic-leukaemia/
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The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
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The MHRA is an executive agency of the Department of Health and Social Care.
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