FDA grants orphan drug designation to dual-target CAR-T for advanced multiple myeloma
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The FDA granted orphan drug designation to GC012F, a novel chimeric antigen receptor T-cell therapy for the treatment of relapsed or refractory multiple myeloma.
GC012F (Gracell Biotechnologies) is an autologous, gene-edited, bispecific CAR-T that targets the B-cell maturation antigen (BCMA) and CD19 proteins on the surface of cancer cells.
The therapy is manufactured using Gracell's proprietary FasT CAR platform, which produces patient-derived therapies within 24 hours followed by a quality-control testing period typical of other CAR T-cell therapies.
“We are very excited about being granted orphan drug designation for the treatment of multiple myeloma by the U.S. FDA, another key milestone in advancing our program globally,” Martina Sersch, MD, chief medical officer of Gracell, said in a company-issued press release. “[Patients with multiple myeloma] are in need of more efficacious and tolerable therapies providing deep and durable responses and ultimately extending progression-free and overall survival.”
GC012F is being evaluated as part of an investigator-initiated phase 1 multicenter trial in China for patients with relapsed or refractory multiple myeloma.
At this year’s virtual ASCO Annual Meeting, researchers presented data from 19 patients who received a single infusion of the therapy. Nearly all patients were heavily pretreated and had high-risk disease per Mayo Stratification for Myeloma and Risk-Adapted Therapy 3.0 criteria.
Median follow-up was 13.8 months (range, 6.1-16.4). Results showed an objective response rate of 94.7%, with 84.2% of patients achieving a minimal residual disease-negative stringent complete response to therapy.
Gracell officials intend to submit an investigational new drug application for GC012F to the FDA by the first half of 2022.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are 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. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
Reference:
Jiang H, et al. Abstract 8014. Presented at: ASCO Annual Meeting (virtual meeting); June 4-8, 2021.
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