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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin
Date Designated: 03/04/2020
Orphan Designation: Treatment of cystinosis
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Novartis Pharmaceuticals Corporation
One Health Plaza
East Hanover, New Jersey 07936-1080
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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