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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: Alpha 1 anti-trypsin (AAT), human alpha 1 anti-trypsin, alpha 1 proteinase inhibitor
Date Designated: 07/25/2023
Orphan Designation: Treatment of Charcot-Marie-Tooth Disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Ageronix SA
4 rue du Tir-au-Canon
Carouge (Geneva) 1227
Switzerland

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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