New treatment for adults with acute lymphoblastic leukaemia
Third CAR T-cell therapy for high-mortality cancer
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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 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.
In its overall assessment of the available data, the Committee for Advanced Therapies (CAT), EMA's expert committee for cell- and gene-based medicines, found that the benefits of Aucatzyl outweighed the possible risks in patients with ALL. The CHMP, EMA’s human medicines committee, agreed with the CAT’s assessment and positive opinion, and recommended approval of this medicine.
Aucatzyl is recommended for a conditional marketing authorisation, one of the EU’s regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need. This type of approval allows the Agency to recommend a medicine for marketing authorisation with less complete data than normally expected, if the benefit of a medicine’s immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.
In order to confirm the safety and efficacy of Aucatzyl, the company 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.
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.
Notes
- The applicant for Aucatzyl is Autolus GmbH.
- Aucatzyl was granted eligibility to PRIME on 25 March 2021 for the treatment of relapsed or refractory B cell acute lymphoblastic leukaemia.
- Aucatzyl was designated as an orphan medicinal product on 13 April 2022 for treatment of acute lymphoblastic leukaemia. Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.