Recordati S.p.A : Carbaglu® Receives Orphan Drug Designation In The U.S. For Organic Acidemias
7/3/2014 5:59:46 AM
MILANO, Italy, July 3, 2014 (GLOBE NEWSWIRE) -- Recordati announces that the U.S. Food and Drug Administration (FDA) has granted approval of Orphan Europe's request for orphan drug designation for the use of Carbaglu® (carglumic acid) in the treatment of organic acidemias (OAs).
The term organic acidemias (OAs) applies to a group of serious life-threatening disorders characterized by excretion of non-amino organic acids in urine. Most organic acidemias (OAs) result from dysfunction of a specific step in amino acid catabolism usually the result of deficient enzyme activity. Classic organic acidemias (OAs) such as isovaleric acidemia (IVA), propionic acidemia (PA) and methylmalonic acidemia (MMA) are autosomal recessive disorders of branched-chain amino acid metabolism (leucine, isoleucine, valine).
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