First FDA approval for new CAR-T therapy

The US Food and Drug Administration (FDA) has approved Autolus’ obecabtagene autoleucel (Aucatzyl), a CD19-directed genetically modified autologous T cell immunotherapy, for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL). The efficacy of obecabtagene autoleucel was evaluated in FELIX, an open-label, multicentre, single-arm trial that enrolled adults with relapsed or refractory CD19-positive B-cell ALL. The major efficacy outcome measures were rate and duration of complete remission (CR) achieved within three months after infusion. Additional outcome measures were rate and duration of overall complete remission which includes complete remission and complete remission with incomplete haematologic recovery (CRi), at any time. Of the 65 patients evaluable for efficacy, 27 patients achieved CR within three months. The median duration of CR achieved within three months was 14.1 months. “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,” said Elias Jabbour, US lead investigator of the FELIX study and Professor of Leukaemia, ALL Section Chief, at the University of Texas MD Anderson Cancer Center, Houston, Texas. “This milestone approval, based on the demonstrated clinical benefit of Aucatzyl, brings new hope for adult patients with relapsed/refractory B-ALL.” Autolus’ first commercial product It is the first CAR-T therapy approved by the FDA with no requirement for a Risk Evaluation Mitigation Strategy (REMS) programme. However, the FDA has added a boxed warning for cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and T cell malignancies to the prescribing information. CRS occurred in 75% and neurologic toxicities occurred in 64%, including ICANS in 24%. “We are so pleased to now be able to offer Aucatzyl, our first commercial product, to adult r/r B-ALL patients in the US,” said Dr Christian Itin, Chief Executive Officer of Autolus. “This milestone is the culmination of many years of hard work, the foundational work by our partners at UCL and the unwavering commitment of our internal team, our external partners and shareholders.” Diana Spencer, Senior Digital Content Editor, DDW