Tag Archive: Orphan Drug

Feb 26

FDA Approves Orphan Drug to Treat Aplastic Anemia

On February 21, 2013, the U.S. Food and Drug Administration (“FDA”) granted orphan drug status to an aplastic anemia treatment. Aplastic anemia is a rare condition where bone marrow does not produce sufficient blood cells or platelets. The treatment, placental expanded (PLX) cells, is sponsored by Pluristem Therapeutics, Inc. You can read more about the …

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May 02

FDA Approves New Orphan Drug (Elelyso) to Treat Gaucher Disease

On May 1, 2012, the U.S. Food and Drug Administration (“FDA”) approved a new orphan drug to treat Gaucher disease. The drug, Elelyso (taliglucerase alfa), is an injection therapy used for enzyme replacement in people with the rare genetic disorder.  Gaucher disease is essentially an enzyme deficiency that causes the buildup of lipids in several …

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