Stem cell researcher Pluristem Therapeutics (NASDAQ: PSTI ) announced that the FDA granted its patented PLacental eXpanded (PLX) cells orphan drug status for the treatment of aplastic anemia, a rare disorder caused by the failure of certain cells in bone marrow to produce red and white blood cells, and platelets. It marks the second time the PLX cells have been given the designation, the first time being in 2011, for the treatment of Buerger's disease.
Conferring orphan drug status on products gives them a seven-year period of exclusivity upon their approval, along with tax credits for certain clinical testing expenses, grants, and other benefits.
Pluristem's CEO Zami Aberman said:
Receiving orphan drug designation for aplastic anemia is an important event for Pluristem as it open pathways for using our PLX cells for additional indications in the field of hematology.
PLacental eXpanded cells are derived from the full-term placenta, which otherwise would be regarded as waste, and then expanded in Pluristem's proprietary bioreactor system that allows for large-scale growth of the cells. Pluristem Therapeutics is a leading developer of placenta-based cell therapies.