Orphan Drug Designation Granted to Immunotherapy for Multiple Myeloma
Posted: Tuesday, October 8, 2019
The U.S. Food and Drug Administration (FDA) has recently granted Orphan Drug designation to the investigational chimeric antigen receptor T-cell therapy CT053 for the treatment of multiple myeloma.
CT053 consists of genetically modified human T cells that target cancer cells containing the B-cell mature antigen protein. CT053 has been part of an exploratory phase I clinical study in China, with a total of 19 of 24 patients with relapsed and refractory multiple myeloma showing a complete response. No event of grade 3 or higher cytokine release syndrome has been reported.
More recently, the FDA has also cleared the investigational new drug application for CT053, allowing it to be tested in the United States.
