Pluristem Therapeutics, a developer of placenta-based cell therapies, is seeking FDA orphan drug status for its PLacental eXpanded (PLX) cells for the treatment of aplastic anemia.
Pluristem earlier received an orphan drug status from the FDA for its PLX cell therapy in the treatment of Buerger's disease and the new filing marks the company's second orphan drug application to the FDA.
Pluristem chairman and CEO Zami Aberman said the pediatric patients afflicted with aplastic anemia exhibited recovery when given PLX cells under compassionate use.
"We are now in the process of establishing an advisory board made up of key opinion leaders in the area of bone marrow transplantation from the United States, Europe and Israel to provide us with valuable insight as we expand our activities in the treatment of the bone marrow diseases and transplantations," Aberman added.
Aplastic anemia is a disorder caused by destruction of blood-forming stem cells (Hematopoietic Stem Cells or HSCs) in the bone marrow.