Obecabtagene autoleucel, sold under the brand name Aucatzyl, is an

anti-cancer medication Chemotherapy (often abbreviated chemo, sometimes CTX and CTx) is the type of cancer treatment that uses one or more anti-cancer drugs ( chemotherapeutic agents or alkylating agents) in a standard regimen. Chemotherapy may be given with a cu ...

used for the treatment of

acute lymphoblastic leukemia Acute lymphoblastic leukemia (ALL) is a cancer of the Lymphocyte, lymphoid line of blood cells characterized by the development of large numbers of lymphoblast, immature lymphocytes. Symptoms may include feeling tired, pale skin color, fever, ...

. It is a

CD19 B-lymphocyte antigen CD19, also known as CD19 molecule ( Cluster of Differentiation 19), B-Lymphocyte Surface Antigen B4, T-Cell Surface Antigen Leu-12 and CVID3 is a transmembrane protein that in humans is encoded by the gene ''CD19''. In human ...

-directed genetically modified autologous

T-cell T cells (also known as T lymphocytes) are an important part of the immune system and play a central role in the adaptive immune response. T cells can be distinguished from other lymphocytes by the presence of a T-cell receptor (TCR) on their ce ...

immunotherapy. The most common side effects include

cytokine release syndrome In immunology, cytokine release syndrome (CRS) is a form of systemic inflammatory response syndrome (SIRS) that can be triggered by a variety of factors such as infections and certain drugs. It refers to cytokine storm syndromes (CSS) and occur ...

, infections-pathogen unspecified, musculoskeletal pain, viral infections, fever, nausea, bacterial infectious disorders, diarrhea, febrile neutropenia, immune effector cell-associated neurotoxicity syndrome, hypotension, pain, fatigue, headache, encephalopathy, and hemorrhage. Obecabtagene autoleucel was approved for medical use in the United States in November 2024.

Medical uses

Obecabtagene autoleucel is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

Side effects

The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) approved prescribing information for obecabtagene autoleucel has a

boxed warning In the United States, a boxed warning (sometimes "black box warning", colloquially) is a type of warning that appears near the beginning of the package insert for certain prescription drugs, so called because the U.S. Food and Drug Administratio ...

for

, immune effector cell-associated neurotoxicity syndrome, and

malignancies. The most common side effects include cytokine release syndrome, infections-pathogen unspecified, musculoskeletal pain, viral infections, fever, nausea, bacterial infectious disorders, diarrhea, febrile neutropenia, immune effector cell-associated neurotoxicity syndrome, hypotension, pain, fatigue, headache, encephalopathy, and hemorrhage.

History

Efficacy was evaluated in FELIX (NCT04404660), an open-label, multicenter, single-arm trial that enrolled adults with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia. Enrolled participants were required to have relapsed following a remission lasting twelve months or less, relapsed or refractory acute lymphoblastic leukemia 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. The major efficacy outcome measures were rate and duration of complete remission 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 hematologic recovery, at any time. Of the 65 participants evaluable for efficacy, 27 participants (42%; 95% confidence interval I 29%, 54%) achieved complete remission within three months. The median duration of complete remission achieved within three months was 14.1 months (95% CI: 6.1, not reached). The US

(FDA) granted the application for obecabtagene autoleucel regenerative medicine advanced therapy (RMAT) and

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designations.

Society and culture

Legal status

Obecabtagene autoleucel was approved for medical use in the United States in November 2024. In May 2025, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regar ...

of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Aucatzyl, intended for the treatment of adults from 26 years of age with relapsed or refractory B cell precursor acute lymphoblastic leukemia. The applicant for this medicinal product is Autolus GmbH. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

Names

Obecabtagene autoleucel is the international nonproprietary name. It is sold under the brand name Aucatzyl.

References

External links

* * Antineoplastic drugs Approved gene therapies CAR T-cell therapy Drugs that are a gene therapy Orphan drugs {{antineoplastic-drug-stub