(By Balaseshan) Pluristem Therapeutics Inc. (NASDAQ:PSTI), a developer of placenta-based cell therapies, said it has filed the necessary documents that the U.S. regulatory authorities grant orphan drug status to its PLacental eXpanded (PLX) cells to treat aplastic anemia.
The filing marks Pluristem's second orphan drug application to the U.S. FDA. The company applied once before, and successfully received in August of 2011, orphan drug status from the FDA for its PLX cell therapy in the treatment of Buerger's disease.
Aplastic anemia is a rare but serious disorder caused by destruction of blood-forming stem cells (Hematopoietic Stem Cells or HSCs) in the bone marrow.
While normally HSCs develop into three types of blood cells, red blood cells, white blood cells and platelets, in aplastic anemia all blood types are deficient, a condition also known as pancytopenia.
The disease is considered an emergency situation where patients are supported either with blood transfusions in anticipation of a bone marrow transplant (BMT) or with drugs that suppress the immune system.
Aplastic anemia patients are included in the bone marrow transplant market estimated at $1.3 billion per year in the U.S. alone.
"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," said Zami Aberman, Chairman and CEO of Pluristem.
PSTI closed Wednesday's regular session at $3.94. The stock has been trading between $1.98 and $5.00 for the past 52 weeks.