The U.S. Food and Drug Administration (FDA) has granted its Regenerative Medicine Advanced Therapy (RMAT) designation to the investigational therapy CT053 for the treatment of relapsed or refractory multiple myeloma.
The RMAT designation is given to regenerative therapies intended to treat, modify, reverse, or cure a serious condition, and for which preliminary clinical data indicates the therapy’s potential to address unmet medical needs for a condition.
The status grants all the benefits of Fast Track and Breakthrough Therapy designations, including early interactions with sponsors and the FDA, which may be used to discuss intermediate or surrogate clinical trial endpoints and support accelerated approval of the therapy.
The designation follows a recently granted orphan drug designation by the FDA and priority medicines (PRIME) eligibility from the European Medicines Agency. It was based on data from an ongoing Phase 1 clinical trial (NCT03975907), which support CT053’s safety and efficacy in people with heavily pre-treated multiple myeloma.
“RMAT eligibility is an important regulatory milestone for CARsgen in the continued development and commercialization of CT053 anti-BCMA CAR T cell therapy,” Zonghai Li, MD, PhD, CEO of CARsgen Therapeutics, which developed the therapy, said in a news release.
“RMAT as well as the (PRIME) eligibility received from the European Medicines Agency (EMA) empower us to collaborate closely with the U.S. FDA and EMA to rapidly advance the CT053 development program toward global regulatory approvals,” he added.
CT053, an investigational CAR T-cell therapy, uses a patient’s own immune T-cells, equipped with a receptor for the BCMA protein. BCMA is present in most multiple myeloma cells, making it a suitable target for cell-based therapies.
After being collected and genetically engineered in the lab, the T-cells are expanded to millions and injected back into the patient’s bloodstream with the aim of eliminating malignant cancer cells while leaving healthy cells unharmed.
In an ongoing, three-site Phase 1 trial (NCT03716856, NCT03302403, and NCT03380039; each number corresponds to a site where the trial is taking place), CT053 is showing promising response rates in a group of 24 patients with heavily treated disease (median of 4.5 lines of therapy).
Results presented in September at the 17th International Myeloma Workshop revealed that 88% of patients experienced a significant reduction in tumor burden with the treatment. These included 79% whose cancer disappeared completely and 67% whose cancer is still in complete response or very good partial response after a median follow-up of 9.8 months.
Cytokine release syndrome — an adverse event of immunotherapies characterized by an excessive activation of the immune system — was observed in 15 patients, but it was mild to moderate in severity.
“The RMAT designation indicates that CT053 has demonstrated potential to address unmet medical needs for patients with [relapsed or refractory multiple myeloma],” Li said. “The designation is a remarkable achievement towards expediting the product development and review of our planned biologics license application (BLA) and will be invaluable to bringing this cutting-edge advance to patients as quickly as possible.”