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FDA Approves Orphan Drug to Treat Aplastic Anemia

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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 requirements for obtaining orphan drug status here. Pursuant to the Orphan Drug Designation program, the FDA provides incentives for the development of products that will diagnose or treat rare diseases. Orphan drug status qualifies a drug sponsor to receive tax credits for clinical testing of…
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FDA Approves New Orphan Drug (Elelyso) to Treat Gaucher Disease

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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 organs, including the liver and kidneys. Approved specifically to replace the missing enzyme (glucocerebrosidase) in patients with a confirmed diagnosis of Type 1 Gaucher disease, the drug underwent testing and approval through the FDA’s orphan drug designation process. Pursuant to the Orphan Drug Act of…
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