Bellicum Pharmaceuticals announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation for the combination of BPX-501 and activator agent rimiducid, as a replacement T-cell therapy for the treatment of immunodeficiency and graft versus host disease (GvHD) after allogeneic hematopoietic stem cell transplant.

A lack of sufficient mature T cells following an allogeneic transplant constitutes an immune deficiency that can lead to infections, viral reactivation and relapse. The addition of BPX-501 as a replacement T-cell therapy corrects this immune deficiency by adding back mature donor T cells without increasing the risk of unmanageable GvHD.

RELATED: Immune Disorders Resource Center 

BPX-501 is currently under investigation in multiple Phase 1/2 clinical studies in adults and children with leukemias, lymphomas, and genetic blood disorders. Preliminary results from these studies demonstrated positive outcomes for BPX-501.

BPX-501 is an adjunct T cell therapy of genetically modified donor T cells incorporating Bellicum’s proprietary CaspaCIDe safety switch. This enables an ability to eliminate BPX-501 alloreactive T cells should severe GvHD occur, and allows for safe stem cell transplantation by adding BPX-501 T cells back in order to speed immune reconstitution and provide control over viral infections.

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