The U.S. Food and Drug Administration (FDA) has approved Autolus Therapeutics' Aucatzyl (obe-cel), a CAR T-cell therapy, for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). This approval marks a significant milestone for patients facing this aggressive blood cancer, which has limited treatment options and poor survival rates. The decision was announced by Syncona, a life science investor and co-founder of Autolus.
The FDA's approval of Aucatzyl was primarily based on data from the pivotal FELIX clinical trial. The trial demonstrated a robust safety profile and significant efficacy. Among the 65 evaluable patients, 63% achieved complete remission. Furthermore, 42% of patients achieved complete remission within three months. The median remission duration was 14.1 months, indicating a durable response to the therapy.
A notable aspect of Aucatzyl's approval is that it is the first CAR T-cell therapy in this category to receive approval without a Risk Evaluation and Mitigation Strategy (REMS) requirement. REMS are FDA-imposed safety programs often mandated for treatments with serious risks. The absence of this requirement underscores the favorable safety profile observed in the FELIX trial.
Addressing Unmet Needs in r/r B-ALL
Relapsed or refractory B-ALL is an aggressive form of leukemia with limited treatment options and poor survival rates. Approximately 8,400 new cases of ALL are diagnosed annually in the US and the European Union, with around 3,000 of those cases being in the relapsed/refractory category. The median survival for adults with r/r B-ALL is only around eight months, highlighting the urgent need for more effective therapies.
Manufacturing and Distribution
Aucatzyl will be manufactured at Autolus' facility in Stevenage, UK, with plans for global distribution. This marks a crucial step in scaling the company's therapeutic offerings and ensuring access to patients in need.
Syncona's Perspective
Chris Hollowood, chief executive officer of Syncona Investment Management, expressed his enthusiasm for the approval, stating, "We are delighted for Autolus to receive FDA approval for its novel CAR T-cell therapy for the treatment of adult ALL. Adult ALL is a devastating disease and Aucatzyl will bring a much-needed new treatment option to patients suffering from the condition. This is a proud moment for Syncona - we co-founded Autolus in 2014 and it is the first company we have supported from the academic bench through to regulatory approval."
