New treatment for adults with acute lymphoblastic leukaemia
EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Aucatzyl (obecabtagene autoleucel) to treat adults from 26 years of age with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (B ALL).
Acute lymphoblastic leukaemia (ALL) is a fast-growing and life-threatening cancer that affects the blood and bone marrow, specifically impacting white blood cells (lymphocytes). Relapsed ALL comes back after treatment, and refractory ALL does not respond to initial treatment. Despite multiple available therapeutic options, this condition is associated with significant mortality and a poor survival rate.
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.
Aucatzyl was supported through EMA’s PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients’ unmet medical needs.
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 opinion adopted by the CHMP is an intermediary step on Aucatzyl’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.