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 Tecartus’s. 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 Aucatzyl’s 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 FDA’s 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.
- SEO Powered Content & PR Distribution. Get Amplified Today.
- PlatoData.Network Vertical Generative Ai. Empower Yourself. Access Here.
- PlatoAiStream. Web3 Intelligence. Knowledge Amplified. Access Here.
- PlatoESG. Carbon, CleanTech, Energy, Environment, Solar, Waste Management. Access Here.
- PlatoHealth. Biotech and Clinical Trials Intelligence. Access Here.
- Source: https://www.biopharmadive.com/news/autolus-fda-approval-aucatzyl-obe-cel-car-t/732543/