Search Orphan Drug Designations and Approvals
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Generic Name: | ataluren |
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Date Designated: | 09/01/2004 |
Orphan Designation: | For use in the treatment of cystic fibrosis resulting fron a nonsense (premature stopcodon) mutation in the cystic bibrosis transmembrane conductance regulatory gene |
Orphan Designation Status: | Designated/Designation Withdrawn or Revoked |
Date Designation Withdrawn or Revoked: | 06/21/2017 |
FDA Orphan Approval Status: | Not FDA Approved for Orphan Indication |
Sponsor: |
PTC Therapeutics, Inc. 100 Corporate Court South Plainfield, New Jersey 07080 United States The sponsor address listed is the last reported by the sponsor to OOPD. |
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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