On 28 October 2019, CARsgen Therapeutics, a clinical-stage biopharma company, announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational CT053 CAR-T cell therapy.
What is CT-053:
CT053 is a fully human anti-BCMA (B Cell Maturation Antigen) autologous chimeric antigen receptor (CAR) T Cell therapy for the treatment of relapsed and/or refractory multiple myeloma.
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising regenerative medicines and advanced therapies, including CAR T cell therapies. The designation includes all the benefits of the FDA’s Fast Track and Breakthrough Therapy designations, providing the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA senior management to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the BLA and other opportunities to expedite development and review.
CAR-T cell therapies are based on the principle of harnessing the innate potential of the immune system to selectively target and destroy diseased cells. These involve isolation of T-cells, which are modified to express a synthetic receptor that enables them to recognize tumor-specific antigens and mount an immune response against cells bearing such antigens. Initial efforts in this domain were focused on the development of a CAR-T therapy against the CD19 receptor found on B-cells.
How many approved CAR-T therapies are available: We currently have 2 approved products, namely KYMRIAH® (Novartis) and YESCARTA® (Gilead Sciences).
How many CAR-T therapies are in development: There are currently more than 530+ CAR-T therapies in development. Below is the distribution of the CAR-T pipeline by the phase of development:
To get more insights about the CAR-T pipeline, check out the detailed report here.