Posted from: Monday, October 05, 2015 - 04:59 PM - Present

Procysbi Approved for Younger Children

August 24, 2015 – The U.S. Food and Drug Administration (FDA) granted approval to Raptor Pharmaceuticals for the use of Procysbi (cysteamine bitrartrate) delayed-release capsules to treat nephropathic cystinosis in children 2 to 6 years of age. Procysbi was originally approved on April 30, 2013 as a cysteine-depleting agent for the treatment of nephropathic cystinosis in adults and children 6 years of age and older. Nephropathic cystinosis is an inherited metabolic lysosomal storage disorder in which the protein cystine builds up in every cell of the body, with cystine crystals accumulating in various tissues and organs that can cause kidney failure, blindness, muscle wasting, and premature death. Untreated cycrinosis is fatal within the first decade of life. Cystinosis is also an orphan condition that affects approximately 500 patients in the U.S. 

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Last Updated Tuesday, October 17, 2017 - 12:17 AM.