Search Orphan Drug Designations and Approvals
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Generic Name: | onasemnogene abeparvovec |
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Trade Name: | ZOLGENSMA |
Date Designated: | 09/30/2014 |
Orphan Designation: | Treatment of spinal muscular atrophy |
Orphan Designation Status: | Designated/Approved |
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. |
Sponsor: |
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. |
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