Aucatzyl Approved for Leukemia

On Nov. 8, 2024, Autolus Therapeutics announced that the US Food and Drug Administration (FDA) approved Aucatzyl^® (obecabtagene autoleucel). Aucatzyl is a CD19 chimeric antigen receptor (CAR) T-cell immunotherapy indicated for treating adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). This is an autologous, split-dose infusion to be administered on days one and ten (+/- two days) with dosing regimens determined by tumor burden. It carries boxed warnings for Cytokine Release Syndrome (CRS), neurologic toxicities including Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and secondary hematological cancers; however, this is the first CAR T approved without a Risk Evaluation Mitigation Strategy (REMS) program. The launch date and cost information are not known at this time. For full prescribing information, please click here.

At a Glance

• Brand Drug: Aucatzyl^® (obecabtagene autoleucel)
• Manufacturer: Autolus Therapeutics
• Date Approved: Nov. 8, 2024
• Indication:  For the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL)
• Dosage Forms Available: Cell suspension for infusion containing a total recommended dose of 410 x 10⁶ CD19 CAR-positive viable T cells supplied in three to five infusion bags
• Launch Date: The launch date isn’t known at this time.
• Estimated Annual Cost: Cost information is not known at this time.
• Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia, is a rapidly progressive form of cancer affecting bone marrow and is characterized by an overproduction of lymphocytes or immature white blood cells. These lymphocytes can originate from B-cells or T-cells. One of the surface antigens presented on B-cells is CD-19, which is the target of Aucatzyl. While ALL starts in the bones, it can quickly spread to the blood, lymph nodes, liver, spleen, central nervous system or testicles.
• In the United States, it is estimated that there will be approximately 6,500 new cases of adult ALL in 2024 and 1,300 deaths. Survival rates remain poor in adult patients with r/r ALL as overall survival is eight months and only 30-40% of adult patients with ALL achieve long-term remission.
• This approval is based on results from the FELIX clinical trial, an open-label, single-arm trial in which adult patients with r/r B-ALL received at least one infusion of Aucatzyl following at least two prior lines of therapy or had relapsed at least three months after allogeneic stem cell transplant. Of the 65 efficacy evaluable patients, 63% achieved overall complete remission while 42% of patients achieved it within three months. The median duration of remission was about 14 months.
• This trial showed the most common adverse events included CRS, neurological toxicities, and secondary malignancies resulting in boxed warnings. CRS is a systemic inflammatory response characterized by low blood pressure and difficulty breathing. The FDA did not require a REMS for Aucatzyl due to low levels of CRS observed in the trial.
• Aucatzyl will compete with Tecartus^® (brexucabtagene autoleucel – Gilead), an intravenous CD19-directed CAR T-cell immunotherapy indicated for the treatment of adult patients with r/r B-ALL; it is given as a one-time dose.