Gamida Cell announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation for NiCord as a treatment for hematopoietic stem cell transplantation (HSCT).
NiCord was previously granted Orphan Drug Designation as a treatment for several hematologic malignancies. It is also the first bone marrow transplant alternative to be granted Breakthrough Therapy status by the FDA.
NiCord is being developed as a universal bone marrow transplant option for patients with high-risk hematologic malignancies. The stand-alone graft, which utilizes the Company’s NAM technology, is a potential option to treat most high-risk patients who do not have a rapidly available, fully matched donor. After a patient is deemed eligible for transplantation with NiCord, an eligible cord blood unit is selected and the manufacturing process begins. During the ~3 weeks of manufacturing, the patient is given a standard conditioning course that lasts ~8 to 11 days.
Compared with unmanipulated cord blood, treatment with NiCord was associated with greater efficacy as well as fewer bacterial and fungal infections and decreased duration of hospital stay. Currently, a Phase 3 study is evaluating NiCord in patients with leukemia and lymphoma in the US, Europe, and Asia.
For more information visit Gamida-cell.com.