FDA Approves Autolus’ New CAR-T Therapy for Aggressive Blood Cancer

In a significant development for cancer treatment, the US Food and Drug Administration (FDA) has approved Autolus Therapeutics’ CAR-T cell therapy for adult patients with relapsed or treatment-resistant B cell precursor acute lymphoblastic leukemia (B-ALL). This approval marks a critical advancement in CAR-T cell therapies targeting aggressive forms of blood cancer.

Autolus’ therapy, a CD19-targeted CAR-T treatment, is priced at $525,000 and joins the market with Gilead’s Tecartus, approved in 2021 for adult B-ALL at a price of around $460,000. While Novartis’ Kymriah is also FDA-approved, it’s limited to B-ALL patients up to age 25. Autolus aims to have 30 treatment centers for the new therapy operational soon after its launch, with plans to expand to 60 centers by the end of 2025.

Safety and Efficacy from the FELIX Trial

The FDA’s approval was based on data from the pivotal FELIX trial, which showed promising outcomes for patients with advanced disease. Among 65 patients with over 5% bone marrow blasts, 63% achieved complete remission, with a remission duration averaging 14.1 months. Importantly, high-grade adverse effects were rare: only 3% experienced severe cytokine release syndrome (CRS), and 7% experienced neurotoxicity events.

No REMS Required, Reflecting Advances in Safety Management

Despite the serious risks of CAR-T therapies, including potential cytokine release syndrome and neurologic toxicities, the FDA chose not to impose a Risk Evaluation and Mitigation Strategy (REMS) for this treatment. This aligns with recent regulatory adjustments to ease the monitoring burden on CAR-T therapy providers, a decision influenced by the healthcare community’s increasing expertise in managing these side effects.

Expanding Access to Life-Saving Therapies

The therapy will be produced at Autolus’ UK-based manufacturing site in Stevenage. It is also under review in the UK and EU, with marketing authorization submissions accepted by both the European Medicines Agency and the UK’s Medicines and Healthcare products Regulatory Agency.

Dr. Elias Jabbour, the US lead investigator of the FELIX study, emphasized the urgency of developing treatments like this: “Adult ALL is an extremely aggressive cancer, and a high unmet need exists in treating patients who relapse and face limited options.”

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