China-based biopharmaceutical firm CARsgen Therapeutics has received regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for CT053 to treat relapsed and refractory multiple myeloma (rrMM).
CT053 is an autologous chimeric antigen receptor (CAR) T-cell therapy, designed as a fully human anti-B Cell Maturation Antigen (BCMA).
The FDA granted RMAT designation based on results from an ongoing Phase I clinical trial in heavily pre-treated patients with multiple myeloma in China.
The RMAT designation is expected to speed-up drug development and review processes for regenerative medicines and advanced therapies such as CAR T-cell treatments.
It also enables FDA guidance on development, surrogate or intermediate endpoints, faster approval and post-approval requirements.
CARsgen Therapeutics CEO Zonghai Li said: “RMAT eligibility is an important regulatory milestone for CARsgen in the continued development and commercialisation of CT053 anti-BCMA CAR T cell therapy.
“The RMAT designation indicates that CT053 has demonstrated the 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.”
In September, CT053 secured PRIME eligibility status from the European Medicines Agency, which should also expedite the development of promising drugs.
Li added: “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 programme toward global regulatory approvals.”
Furthermore, CT053 obtained investigational new drug (IND) approval and orphan drug designation from the FDA and Health Canada authorisation for its clinical trial application (CTA).