Aucatzyl

Aucatzyl is indicated for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B cell precursor acute lymphoblastic leukaemia (B-ALL). Key efficacy findings include an Overall Remission Rate (CR+CRi) of 75.0% and 87.5% in different cohorts. The Overall Response Rate (ORR) in Cohort IIA (infused set) was 76.6%, with a Complete Remission (CR) rate of 55.3% in the same cohort. The KM estimate of probability of ongoing event-free remission at 12 months in patients with high tumour burden was 46.2%, decreasing to 30.8% at 21 months. CAR T cell persistency in responders (FELIX study) was 75.0%, and 88% in ongoing remission (ALLCAR19 study) with a median follow-up of 36 months. The maximum CAR T persistency observed was 30 months.

Regarding safety, the incidence of Any Grade CRS (Total) was 70.0%, with Grade ≥3 CRS for high disease burden regimen at 2.8%. The incidence of Grade ≥3 ICANS (Total) was 5.8%. Positive humoral immunogenicity findings post-infusion were observed in 2 cases. The drug received Orphan medicinal product designation on 13 April 2022 and PRIME eligibility on 25 March 2021. A CHMP positive opinion was issued on 22 May 2025.