Stem cell therapy developer Gamida Cell has been awarded orphan drug status by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for leukemia treatment NiCord. The investigational drug treats acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), Hodgkin lymphoma and myelodysplastic syndrome (MDS). Gamida Cell intends to file for NiCord orphan drug status with the EMA for other indications as well.
Gamida Cell president and CEO Dr. Yael Margolin said, "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."
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 US and 10 years in the EU.
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.
Published by Globes [online], Israel business news - www.globes-online.com - on January 6, 2015
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