Brexucabtagene Autoleucel

US regulators gave the green light to Autolus Therapeutics’ cell therapy for adult B cell precursor acute lymphoblastic leukemia, or B-ALL, that has returned or is not responding to prior treatment. Autolus will market its CD19-targeted cell therapy as Aucatzyl, according to the FDA’s announcement Friday, which came about a week earlier than the agency’s decision deadline. Autolus did not immediately disclose the list price for Aucatzyl, which is known generically as obecabtagene autoleucel, or obe-cel. The US approval lands Aucatzyl in the same market as Gilead’s Tecartus, which was approved for B-ALL in 2021. Like other cell therapies, Aucatzyl comes with a boxed warning for cytokine release syndrome and neurologic toxicities, as well as secondary blood cancers. The warning notes broadly that “T-cell malignancies have occurred” with CAR-T cell therapies, though like Tecartus, no cases of secondary cancers have been reported from Aucatzyl. However, the FDA did not require a special safety, or REMS, program for Aucatzyl. The agency earlier this year loosened safety reporting requirements for CAR-T therapies, a move the FDA said was to minimize burden on the healthcare system, and that came at a time when physicians have said they are more used to treating the side effects of CAR-T therapies. Notably, Aucatzyl’s FDA label cited efficacy data from a subgroup of patients in Autolus’ pivotal FELIX study. The agency focused on a 65-patient cohort with morphological disease and > 5% blasts in the bone marrow after screening. Of those patients, 63% achieved complete remission (at any time). By comparison, Autolus had reported that in a pooled analysis of 127 patients, 78% of patients achieved complete remission. Autolus will manufacture Aucatzyl at its commercial manufacturing site in Stevenage in the UK. The cell therapy is also under review in the UK and EU.

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. '