Gamida Cell Orphan Status for Transplants
Gamida Cell, a developer of cell therapy technologies and products for transplantation and adaptive immune therapy, announced that orphan drug designation has been granted by the FDA for its investigational medicinal product NiCord. The drug is used for the treatment of acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), Hodgkin lymphoma and myelodysplastic syndrome (MDS). The FDA orphan drug designation coincides with the positive opinion of the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) regarding NiCord as a treatment for AML. Gamida Cell intends to file for NiCord orphan drug status with the EMA for other indications as well. NiCord is derived from a single cord blood unit which has been expanded in culture and enriched with stem cells using Gamida Cell’s proprietary NAM technology. It is currently being tested in a Phase I/II study as an investigational therapeutic treatment for hematological malignancies such as leukemia and lymphoma. In this study, NiCord is being used as the sole stem cell source.