Israel’s 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.
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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. Click here for more information.
“Receipt of orphan drug status for NiCord in the US and Europe advances Gamida Cell’s commercialization plans a major step further, as both afford significant advantages. We very much appreciate the positive feedback and support of the FDA and EMA and look forward to continuing what has been a very positive dialogue with these important agencies, ” said Gamida Cell president and CEO Dr. Yael Margolin.
The FDA and EMA grant an orphan drug designation to promote the development of products that demonstrate promise for the treatment of rare diseases or conditions. Orphan drug designation provides for various regulatory and economic benefits, including seven years of market exclusivity in the U.S. and 10 years in the EU.
Gamida Cell says that it has a pipeline of products in development with a potential to treat a wide range of conditions including blood cancers, non-malignant hematological diseases such as sickle cell disease and thalassemia, neutropenia and acute radiation syndrome, autoimmune diseases, solid tumors and genetic metabolic diseases as well as conditions that can be helped by adaptive immune therapy such as solid and blood cancers refractory to chemotherapy. Gamida Cell’s therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture.