Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, has announced that orphan drug designation has been granted by the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) regarding NiCord as a treatment for haematopoietic stem cell transplantation, also commonly known as bone marrow transplantation (BMT).
The EMA grants an orphan drug designation to promote the development of products that demonstrate promise of significant benefit for the treatment of rare diseases. Products receiving orphan drug designation are eligible to receive various regulatory and economic benefits, including 10 years of market exclusivity in the EU.
Gamida Cell was previously granted orphan drug designation for NiCord by the EMA for acute myeloid leukemia (AML), and by the US Food and Drug Administration (FDA) for the treatment of several hematologic malignancies including AML, acute lymphoblastic leukemia (ALL), Hodgkin’s lymphoma, myelodysplastic syndromes (MDS) and chronic myelogenous leukemia (CML). Gamida Cell also received FDA Breakthrough Therapy Designation for NiCord in blood cancers.
NiCord is currently being studied in an international, multi-center, phase 3 registration study as a graft for bone marrow transplantation for patients with blood cancer who do not have a rapidly available fully matched donor. The Company announced last month that the first patient in the study had been transplanted.