CAR-T cell therapy granted RMAT for relapsed/refractory multiple myeloma

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CARsgen Therapeutics Co Ltd’s investigational CT053 CAR-T cell therapy.

CT053 is a fully human anti-BCMA (B Cell Maturation Antigen) autologous CAR T cell therapy for the treatment of relapsed and/or refractory multiple myeloma (rrMM).

“RMAT eligibility is an important regulatory milestone for CARsgen in the continued development and commercialisation of CT053 anti-BCMA CAR T cell therapy,” said Zonghai Li, MD, PhD, Chief Executive Officer, CARsgen. “The RMAT designation indicates that CT053 has demonstrated potential to address unmet medical needs for patients with rrMM. 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.

“RMAT as well as the PRIority MEdicines (PRIME) eligibility received from the European Medicines Agency (EMA) empower us to collaborate closely with the US FDA and EMA to rapidly advance the CT053 development program toward global regulatory approvals.”

The designation was based on clinical data from an ongoing CT053 Phase I study in heavily pre-treated multiple myeloma patients in China. Updated data from CT053 will be presented on 9 December.

The CT053 anti-BCMA CAR-T programme has also received Investigational New Drug (IND) clearance and Orphan Drug designation from the FDA and authorisation of its Clinical Trial Application (CTA) from Health Canada.