Pluristem seeks FDA orphan drug status for bone marrow treatment

The company's placental stem cells successfully treated an Israeli girl earlier this year, after bone marrow transplants failed.

Pluristem Therapeutics Ltd. (Nasdaq:PSTI; DAX: PJT: PLTR) is preparing to apply to the US Food and Drug Administration (FDA) for orphan drug status for its PLacental eXpanded (PLX) cells for the treatment of aplastic bone marrow.

Aplastic bone marrow is a disease in which bone marrow greatly decreases or stops production of blood cells and which strikes five to ten people in every million. Pluristem, a developer of placenta-based cell therapies, received orphan drug status PLX cell therapy in the treatment of Buerger's disease in August 2011.

Earlier this year, Pluristem reported that its PLX treatment saved the life of a seven year-old girl in Israel suffering from aplastic bone marrow and who had undergone two failed bone marrow transplants. Her doctors injected the PLX cells intramuscularly, following which she experienced a reversal of her condition with a significant increase in her red blood cells, white blood cells and platelets.

Pluristem chairman and CEO Zami Aberman said, "We are very encouraged by the therapeutic results we've seen for our PLX cells in the treatment of aplastic anemia and we intend to pursue every avenue to bring it to market for this indication as swiftly as possible and to extend the indication to other clinical situations involving bone marrow deficiencies." He added that obtaining orphan drug status in the US would help the company achieve full FDA approval and that the company intends to apply for orphan drug status in Europe and elsewhere."

Orphan drug status carries multiple potential benefits, including the possibility of an expedited regulatory process, availability of grant money, certain tax credits and seven years of market exclusivity.

Pluristem's share price rose 0.1% by mid-afternoon on the TASE today to NIS 12.66, after rising 5.6% on Nasdaq yesterday to $3.38, giving a market cap of $151 million, although it shed the gain in premarket trading today.

Published by Globes [online], Israel business news - www.globes-online.com - on July 23, 2012

© Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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