Pluristem Therapeutics, Inc. (PSTI) recently provided an update on its efforts to gain orphan drug status for its PLacental eXpanded (PLX) cells in the treatment of aplastic anemia. The company had submitted its application seeking orphan drug status in the US in August 2012.
While the FDA said that aplastic anemia is indeed a rare disease with fewer than 200,000 people suffering from it in the US, the agency has asked the company to provide it with additional information and data. The FDA intends to analyze the data and information before granting orphan drug status to PLX cells for aplastic anemia.
Meanwhile, Pluristem has moved PLX cells into a phase I/II study which is being conducted to evaluate its safety and efficacy in the treatment of muscle injury. Two doses of PLX cells will be compared to placebo.
We currently have a Neutral recommendation on Pluristem, which carries a Zacks #3 Rank (short-term Hold rating). The company is focused on the development and subsequent commercialization of cell therapy production technologies and products. Other companies working in this area include Osiris Therapeutics (OSIR).
We note that on being provided with orphan drug status by the FDA, the PLX cell therapy for treating aplastic anemia patients will enjoy multiple benefits including seven years of marketing exclusivity (from the day the candidate is cleared by the FDA) in the US for the indication.
We remind investors that Pluristem enjoys orphan drug status for using PLX cells to treat patients suffering from Buerger's disease.
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