The US Food and Drug Administration (FDA) has approved Autolus Therapeutics’ Aucatzyl (obecabtagene autoleucel) to treat acute lymphoblastic leukaemia (ALL).
The CD19-directed CAR-T cell therapy has been specifically authorised for use in adults with relapsed or refractory B-cell precursor ALL.
Aucatzyl is now the first CAR-T therapy approved by the US regulator with no requirement for a risk evaluation mitigation strategy programme, which is used for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks.
Approximately 8,400 adults are diagnosed every year in the US and EU with ALL, an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system and other organs.
Survival rates remain very poor for adults in the relapsed/refractory setting, with a median overall survival of eight months.
Aucatzyl is designed to “overcome the limitations” in clinical activity and safety compared to current CD19 CAR T-cell therapies, according to Autolus.
The FDA’s decision on the therapy was supported by positive data from the phase 2 FELIX trial, which enrolled B-cell precursor ALL patients who had relapsed following a remission lasting 12 months or less, had relapsed or refractory ALL following two or more prior lines of systemic therapy, or disease that was relapsed or refractory three or more months after allogeneic stem cell transplantation.
Results showed that 42% of patients achieved the major efficacy outcome of complete remission within three months of Aucatzyl infusion, with a median duration of remission of 14.1 months.
FELIX lead investigator, Elias Jabbour, University of Texas MD Anderson Cancer Center, said: “Adult ALL is an extremely aggressive cancer, and there is a high unmet medical need that exists in the treatment of patients with this disease once they relapse, where historically they suffer from poor outcomes.
“This milestone approval, based on the demonstrated clinical benefit of Aucatzyl, brings new hope for adult patients with relapsed/refractory B-ALL.”
Marketing authorisation applications for Aucatzyl in adult relapsed/refractory ALL are being reviewed by regulators in both the EU and UK.