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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: voretigene neparvovec-rzyl
Trade Name: LUXTURNA
Date Designated: 03/18/2015
Orphan Designation: Treatment of inherited retinal dystrophy due to biallelic RPE65 gene mutations
Orphan Designation Status: Designated/Approved
Spark Therapeutics, Inc.
3737 Market Street
Suite 1300
Philadelphia, Pennsylvania 19104
United States

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

Marketing approved:

1 Generic Name: voretigene neparvovec-rzyl
Trade Name: LUXTURNA
Marketing Approval Date: 12/19/2017
Approved Labeled Indication: an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells determined by a treating physician
Exclusivity End Date: 12/19/2024 
Exclusivity Protected Indication* :  Treatment of patients with confirmed biallelic RPE65 mutation-associated retinal distrophy. Patients must have viable retinal cells determined by a treating physician.
*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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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