FDA Approves Autolus' Aucatzyl for Acute Lymphoblastic Leukemia

The US Food and Drug Administration (FDA) has recently granted approval for Autolus Therapeutics' Aucatzyl (obecabtagene autoleucel) for the treatment of acute lymphoblastic leukaemia (ALL). This CD19-directed CAR-T cell therapy is specifically indicated for adults experiencing relapsed or refractory B-cell precursor ALL.

The approval of Aucatzyl marks a significant development as it is the first CAR-T therapy endorsed by the US regulator without the necessity of a risk evaluation mitigation strategy programme. Such programmes are generally mandated for certain medications with severe safety concerns to ensure that their benefits outweigh the associated risks.

Every year, approximately 8,400 adults in the US and EU are diagnosed with ALL, a particularly aggressive form of blood cancer. This malignancy can extend to various organs, including the lymph nodes, spleen, liver, and central nervous system. The prognosis for adults with relapsed or refractory ALL is often grim, with median overall survival rates at just eight months.

Autolus has stated that Aucatzyl is engineered to address the limitations seen in clinical activity and safety with existing CD19 CAR T-cell therapies. The FDA's decision was influenced by positive results from the phase 2 FELIX trial. This trial included patients with B-cell precursor ALL who had relapsed after a remission lasting a year or less, had relapsed or refractory ALL following at least two prior systemic therapies, or had relapsed/refractory disease three or more months after an allogeneic stem cell transplant.

The findings from the FELIX trial were promising. They indicated that 42% of patients achieved complete remission within three months of receiving Aucatzyl, with the median duration of remission being 14.1 months. Dr. Elias Jabbour from the University of Texas MD Anderson Cancer Center, who was the lead investigator for FELIX, emphasized the critical need for effective treatments for adult ALL patients who relapse, as they typically face poor outcomes.

Dr. Jabbour remarked, "Adult ALL is a highly aggressive cancer, and there is a significant unmet medical need for treatments in patients who relapse, as historically, they have experienced poor results. This milestone approval, based on the clinical benefit demonstrated by Aucatzyl, brings new hope for adult patients with relapsed/refractory B-ALL."

In addition to the FDA approval, marketing authorisation applications for Aucatzyl are currently under review by regulatory authorities in both the EU and the UK. This development signifies a potential advance in the treatment landscape for adult patients suffering from this challenging and aggressive form of leukaemia.