Autolus Therapeutics has secured US Food and Drug Administration (FDA) approval for Aucatzyl (obecaktagene autoleucel), its chimeric antigen receptor (CAR)-T cell therapy for adult patients with relapsed or refractory B cell precursor acute lymphoblastic leukaemia (B-ALL).
The approval follows a heightened regulatory focus on CAR-T cell therapy safety this year. In April, the FDA mandated boxed warnings for these therapies due to rare cases of T cell malignancies. Aucatzyl’s labelling includes these standard warnings for risks associated with cytokine release syndrome (CRS) and neurologic toxicities.
Go deeper with GlobalData
The FDA’s approval decision was based on data from the FELIX trial (NCT04404660), which evaluated the therapy in adult patients with relapsed or refractory B-ALL. In a subgroup of 65 patients with morphological disease and over 5% bone marrow blasts, Aucatzyl demonstrated a 63% overall complete remission (CR). The main efficacy outcome — CR within three months — was achieved in 42% of patients, with a median duration of remission of 14.1 months.
Aucatzyl showed a relatively low incidence of severe adverse events in the FELIX trial, with only 3% of patients experiencing Grade 3 CRS and 7% reporting Grade 2 or 3 immune effector cell-associated neurotoxicity syndrome (ICANS). There were no Grade 4 or 5 CRS events.
Despite the risks, the FDA did not require a risk evaluation and mitigation strategy (REMS) programme for Aucatzyl, a decision in line with recent regulatory changes aimed at easing CAR-T therapy monitoring requirements. In June, the FDA removed a requirement for clinicians to report serious cases of the two most common side effects as part of its REMS programme for Gilead’s treatments Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel).
REMS programmes are typically implemented after drug approval when the FDA determines that a medication needs to be closely monitored.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataWith this approval, Aucatzyl enters a market that includes Gilead’s Tecartus, which gained FDA approval for B-ALL in 2021. Autolus has not yet disclosed the list price for Aucatzyl, which is also under review for market authorisation in the UK and European Union, with submissions to the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) accepted in 2024. Production for Aucatzyl will take place at Autolus’s manufacturing facility in Stevenage, UK.
In the announcement accompanying the approval, Dr. Elias Jabbour, US lead investigator of the FELIX study and professor of leukaemia, ALL Section Chief, at The University of Texas MD Anderson Cancer Center, Houston, Texas said: “Adult ALL is an extremely aggressive cancer, and there is a high unmet medical need that exists in the treatment of patients with this disease once they relapse, where historically they suffer from poor outcomes.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
