The Gamida Cell-Teva Joint Venture announced today that orphan designation was granted by the European Commission for the product the venture is developing. The product in question is StemEx, for the treatment of acute lymphoblastic leukemia and acute myeloid leukemia.
StemEx already has orphan drug status in the US. The FDA granted it the designation in March 2005 for use as hematopoietic support in patients with relapsed or refractory hematologic malignancies (cancer of the blood system) who are receiving high dose therapy.
“This is very important news. Having an orphan drug designation for StemEx in the US and now in Europe affords an impressive array of financial and product development benefits. Today’s news moves StemEx one step closer to market launch,” said Gamida Cell CEO Dr. Yael Margolin.
Under EU regulations, orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.
The benefits of orphan drug status include, among other things, market exclusivity in the European Union for 10 years from the date of the orphan medicinal product designation, and an EMEA fee-reduction for the drug.
StemEx is a graft of expanded stem/progenitor cells, derived from a single unit of umbilical cord blood and transplanted in combination with non expanded cells from the same unit. It is currently being researched as an alternative to a bone marrow transplant, for patients with certain blood cancers, in an advanced Phase III, pivotal study called ExCell now in progress at cord blood transplantation clinical sites in the US, Europe and Israel.
Published by Globes [online], Israel business news - www.globes.co.il - on March 11, 2009
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