FDA Grants Orphan Drug Designation to BCMA/CD19-Targeting CAR-T Therapy for Multiple Myeloma Treatment

By Adam Hochron, Staff Writer
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The FDA granted orphan drug designation to GC012F, an investigational dual-targeting BCMA/CD19 CAR-T therapy,  for the potential treatment of multiple myeloma, according to a press release from the manufacturer.

GC012F is currently being evaluated in a phase 1 trial in China. Researchers presented data on the use of GC012F at medical conferences earlier this year. 

One presentation at ASCO 2021 Annual Meeting reported data on the use of GC012F as a treatment for patients with relapsed/refractory multiple myeloma. The trial included 19 patients, with an overall response rate of 94.7%, according to a press release about the trial. All of the patients achieved a paraprotein reduction, with all but one achieving a 100% reduction. At the cutoff date for the data, the median time to follow-up was 13.8 months, while the median duration of response had not been reached. 

In the most recent release, Martina Sersch, MD, PhD, Chief Medical Officer of Gracell, noted that GC012F “has demonstrated fast, deep, and durable responses” in patients with relapsed/refractory multiple myeloma enrolled in the ongoing trial. 

“We are very excited about being granted orphan drug designation for the treatment of multiple myeloma by the US FDA,” Sersch said in the release. “Multiple myeloma patients are in need of more efficacious and tolerable therapies providing deep and durable responses and ultimately extending progression-free and overall survival.” 


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