Vivacta Bio Receives FDA Orphan Drug Designation for Autoimmune Hemolytic Anemia Therapy
Vivacta Bio and Grit Bio announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to GT801, an investigational therapy for autoimmune hemolytic anemia.
Vivacta Biotechnology (Shanghai) Co., Ltd. and Beijing Grit Biotherapeutics Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to GT801, Vivacta Bio's lead investigational product, for the treatment of autoimmune hemolytic anemia (AIHA).
GT801 is an investigational in vivo CAR-T therapy that utilizes mRNA delivered via lipid nanoparticles (LNP). This approach aims to engineer CAR-T cells directly within the patient's body to target and eliminate CD19-positive B cells. The therapy is designed to simplify treatment compared to traditional ex vivo CAR-T methods, which involve cell isolation, expansion, and reinfusion.
AIHA is an autoimmune disorder characterized by the body's destruction of red blood cells. The press release notes that some patients experience inadequate disease control despite multiple lines of therapy, highlighting the need for novel treatment options.
Preliminary clinical results for GT801 have shown promise. Two patients with AIHA treated with GT801 achieved complete responses, with observed increases in hemoglobin levels and depletion of B cells. The ODD in the U.S. provides incentives, such as tax credits and exemption from marketing application user fees, to support the further development of the therapy.