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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- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- Regular Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
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