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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name:  Single stranded adeno-associated virus vector encoding beta-galactosidase-1
Trade Name:  N/A
Date Designated:  11/14/2019
Orphan Designation:  Treatment of GM1 gangliosidosis
Orphan Designation Status:  Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Marketing Approval Date:  N/A
Approved Labeled Indication:  
Exclusivity End Date:    N/A
Axovant Sciences
11 Times Square, 33rd Floor
New York,, New York 10036

The sponsor address listed is the last reported by the sponsor to OOPD.