Aplastic anemia is a rare but serious disorder with a prevalence of less than 200,000 in the US. The disease is caused by the failure of hematopoietic stem cells in the bone marrow to produce red blood cells, white blood cells and platelets. The disease is considered an emergency and patients are supported with blood products in anticipation of a bone marrow transplant or drugs that suppress the immune system.
This is Pluristem's second orphan drug designation from the FDA, after the company obtained the status for PLX cells for the treatment of Buerger's disease in August 2011.
"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," said Pluristem chairman and CEO Zami Aberman.
Pluristem has established clinical advisory board of bone marrow transplantation experts from the US, Europe and Israel to advise the company on how to expand its treatments of bone marrow diseases and transplantations.
Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983, including seven years marketing exclusivity, a tax credit for certain clinical testing expenses, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.
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