FDA Regenerative Medicine Advanced Therapy designation for P-BCMA CAR T-cell therapy

The US Food and Drug Administration (FDA) has granted the P-BCMA-101 CAR T-cell therapy the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of relapsed/refractory multiple myeloma (MM). The decision was based on preliminary results of a phase I clinical trial, whose primary outcome is to define the maximum tolerated dose of P-BCMA-101 based on dose-limiting toxicities.

P-BCMA-101 is an autologous CAR T-cell product, which targets the BCMA (B-cell maturation antigen), a protein highly expressed on the cell surface of MM cells. The RMAT designation is authorized for a drug that is defined as a cell therapy, intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and which is supported by preliminary clinical evidence indicating that it has the potential to address unmet medical needs for such a disease or condition.

The RMAT process allows companies to develop regenerative medicine therapies (including cell therapies) and interact with the US FDA frequently and at a very early stage of a clinical trial. RMAT-products may be granted priority review and accelerated approval.