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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: recombinant, replication incompetent adeno-associated virus viral vector platform with a serotype 8 capsid and a cDNA encoding the human BBS10 gene (AAV8-RK-BBS10)
Date Designated: 10/11/2024
Orphan Designation: treatment of Bardet-Biedl syndrome (BBS)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
MeiraGTx UK II Ltd
450 East 29th Street, 14th Floor
New York, New York 10016
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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