Cellectis Receives FDA RMAT Designation for Leukemia Therapy Candidate
The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Cellectis' lasme-cel cell therapy candidate. The designation is for the treatment of acute lymphoblastic leukemia.
Cellectis announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to lasmecabtagene timgedleucel (lasme-cel). This allogeneic CAR-T cell therapy candidate targets CD22 and is intended for patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).
The RMAT designation acknowledges the potential of lasme-cel to address the unmet medical need in this patient population. The designation is supported by preliminary data from the Phase 1 BALLI-01 trial, which indicated promising efficacy and a manageable safety profile.
Cellectis is scheduled to present final Phase 1 data from the BALLI-01 trial at the European Hematology Association (EHA) Congress. The company stated that the RMAT designation for its allogeneic CAR-T therapy strengthens its discussions with the FDA as it advances lasme-cel through its pivotal program.
The pivotal Phase 2 BALLI-01 trial is currently enrolling patients who meet specific eligibility criteria. Lasme-cel is being developed as an "off-the-shelf" allogeneic CAR-T cell therapy.