（9/12/2016）FDA批准孤儿药CD4CAR（Chimeric Antigen Receptor–engineered T cells directed against the target protein CD4）用于治疗CD4阳性的外周T细胞淋巴瘤。CD4CAR是第一个被FDA批准的CART细胞治疗药物。

FDA Grants Orphan Drug Designation to CD4CAR for the Treatment of Peripheral T-Cell Lymphoma

September 10, 2016

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for chimeric antigen receptor–engineered T cells directed against the target protein CD4 (CD4CAR) for the treatment of peripheral T-cell lymphoma.

The Orphan Drug designation program provides orphan status and associated development incentives to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

About CD4CAR

CD4CAR is in development for CD4–positive T-cell malignancies.The novel CD4-specific chimeric antigen receptor–engineered T cells are properly matched allogeneic human T cells engineered to express an anti–CD4scFV antibody domain. An initial phase I clinical study is being planned through collaboration between iCell Gene Therapeutics; the National Institutes of Health; Indiana Clinical and Translational Sciences Institute; Stony Brook Hospital; and the Blood and Marrow Transplantation Division and the Clinical Trial Research Unit at James Graham Brown Cancer Center at University of Louisville.

Peripheral T-Cell Lymphoma

Although there are clinical development programs ongoing with CAR T cells for CD19-positive hematologic malignancies, CD4-positive peripheral T-cell lymphomas have not been targeted by a CAR therapy in a human trial. They account for 10% to 15% of all non-Hodgkin lymphomas (NHLs) and are more difficult to treat in comparison to B-cell NHLs.

Furthermore, and with few exceptions, T-cell NHLs have poorer outcomes, lower response rates, shorter times to progression, and shorter median survival in comparison to B-cell NHLs. As a result, the standard of care for peripheral T-cell lymphoma is not well established, and the only potential curative regimen is bone marrow transplant. However, not only is bone marrow transplant poorly tolerated, it is not an option for a significant subset of patients with resistant disease. ■