EMA Greenlights Aucatzyl for Adult ALL

At its May 2025 meeting, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) gave a conditional marketing authorization in the European Union for obecabtagene autoleucel (Aucatzyl) to treat adults from 26 years of age with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (B ALL).

A conditional marketing authorization is granted to a medicinal product that fulfils an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required.

B ALL is a fast-growing and life-threatening cancer. Despite multiple available therapeutic options, it is associated with significant mortality and a poor survival rate.

Aucatzyl, whose active substance is obecabtagene autoleucel, is an antineoplastic cell and gene therapy. The autologous immunotherapy consists of the patient’s own T cells engineered to express a chimeric antigen receptor that recognizes and binds to CD19 on target cells. This results in activation of the immunological effect of the T-cell releasing inflammatory cytokines and chemokines, killing CD19-expressing cells. 

The CHMP said that the benefits of obecabtagene autoleucel were its ability to induce remission with a relevant duration in adults with relapsed or refractory acute lymphoblastic leukemia.

Treatment Achieved Durable Response 

The positive decision by the CHMP was based on the results of the FELIX study, a single-arm, open-label phase 1b-2 multicenter study of obecabtagene autoleucel in adults aged 18 years and over with relapsed or refractory B ALL.

In the study, around 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.

Aucatzyl will be available as a 410 x 10⁶ cells dispersion for infusion. 

The drug’s most common side effects include cytokine release syndrome, infections, musculoskeletal pain, pyrexia, pain, nausea, diarrhea, headache, fatigue, and hemorrhage.

To confirm the safety and efficacy of Aucatzyl, the manufacturer has been requested to submit long-term follow-up results of the FELIX study and to conduct a non-interventional study based on a patient registry.

In the meantime, in its overall assessment of the available data, the Committee for Advanced Therapies — the EMA’s expert committee for cell- and gene-based medicines — found that the benefits of Aucatzyl outweighed the possible risks in patients with ALL, and thus the marketing authorization was granted.

Dr Rob Hicks is a retired NHS doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online. He is based in the UK.