Aucatzyl New treatment for Adults with Acute Lymphoblastic Leukaemia
On 22 May 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorization in the EU for Aucatzyl (obecabtagene autoleucel), a Autolus GmbH CAR T-cell therapy for adults aged 26 and older with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (B ALL), a fast-growing and life-threatening cancer with poor survival rates.
Aucatzyl is a genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy, a type of personalised cancer immunotherapy that is based on collecting and modifying the patient's own immune cells to treat their cancer. The modified T cells attach to and kill the cancer cells, thereby helping to clear the cancer from the body.
The recommendation is based on the results of a single-arm, open-label trial (FELIX study) in 113 patients. About 64% of patients had a durable response (a period without disease signs or symptoms after treatment) with a median duration of 14 months. Around 49% showed a complete response, meaning the signs of cancer disappeared.
The most common observed side effects include cytokine release syndrome (a potentially life-threatening condition that can cause high fever, vomiting, shortness of breath, pain, and low blood pressure), immune effector cell-associated neurotoxicity syndrome (a condition that includes problems with use of language, seizures, headache, hallucinations, and mental confusion), and infections. Monitoring and mitigation strategies for these side effects are described in the product information and in the risk management plan.
Aucatzyl received support through EMA's PRIME scheme for unmet medical needs, and its approval is conditional pending long-term follow-up data and further studies, with final EU-wide authorization pending European Commission decision.
Detailed recommendations for the use of this product will be described in the Summary of product characteristics, which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.