Brexucabtagene Autoleucel

Autolus’ Aucatzyl is a B-lymphocyte antigen CD19 CAR-T cell therapy. Credit: NDAB Creativity via Shutterstock. 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. 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. With 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. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Dive Brief:The Food and Drug Administration on Friday approved a new cell therapy for a type of leukemia. Called Aucatzyl, the CAR-T was developed by U.K.-based Autolus Therapeutics to treat people with B-cell precursor acute lymphoblastic leukemia whose disease has progressed following treatment with chemotherapy and other medicines.Aucatzyl will compete with Gilead Sciences Tecartus, which won approval in the same condition in 2021. The Gilead cell therapy had sales of $305 million in the first nine months of 2024, but Autolus executives claim its drug could outperform Tecartus because it was designed to cause fewer side effects.Autolus set a list price of $525,000 on the therapy, 11% higher than Tecartuss. The company said it has 30 treatment centers in the U.S. ready to begin treatment cycles with Aucatzyl, covering around 60% of patients. Another 30 treatment centers could be ready by end of 2025.Dive Insight:CAR-T therapy, which became available with the launch of Novartis Kymriah seven years ago, has transformed treatment for people with certain types of blood cancers like advanced lymphoma, leukemia and multiple myeloma. The one-time treatments, which consist of patients own cells re-engineered to attack diseased cells, can induce long-lasting remissions.But they are expensive and burdensome, requiring patients to undergo chemotherapy before receiving them. The process of withdrawing cells, manufacturing the treatment and then re-infusing it can take several weeks. And a large share of patients experience significant immune and neurological side effects.Autolus designed its cells to be more similar to naturally occurring T cells, so as to increase the number of active cells after infusion as well as to reduce side effects. Aucatzyl is the first CAR-T therapy approved without a risk mitigation plan, which for others requires doctors to have treatments at hand to combat immune-related side effects and close monitoring by infusion centers.Executives justified Aucatzyls price premium over Tecartus based on its design. We believe this pricing reflects the clinical evidence and benefit we have observed to date, as well as the differentiated safety profile, which delivers meaningful economic benefits to the healthcare system, said Christopher Vann, Autolus chief operating officer, on a Monday conference call with analysts.In a note written prior to that call, Matt Phipps, an analyst with William Blair, had estimated peak sales of $300 million for Aucatzyl based on a presumed price of $450,000.The FDAs approval was based on a clinical trial called Felix, in which 63% of trial volunteers who could be evaluated for treatment efficacy experienced a complete remission. The treatment also had a low side effect rate, with 3% of patients experiencing severe cases of an immune reaction called cytokine release syndrome. '