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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name: onasemnogene abeparvovec
Trade Name: ZOLGENSMA
Date Designated: 09/30/2014
Orphan Designation: Treatment of spinal muscular atrophy
Orphan Designation Status: Designated/Approved
AveXis, Inc.
2275 Half Day Road
Suite 200
Bannockburn, Illinois 60015
United States

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

Marketing approved:

1 Generic Name: onasemnogene abeparvovec
Trade Name: ZOLGENSMA
Marketing Approval Date: 05/24/2019
Approved Labeled Indication: Indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Exclusivity End Date: 05/24/2026 
Exclusivity Protected Indication* :  Indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.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