Proper Name: voretigene neparvovec-rzyl
Trade Name: LUXTURNA
Manufacturer: Spark Therapeutics, Inc.
- Is 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 as determined by the treating physician(s).
- Package Insert - LUXTURNA
- Demographic Subgroup Information - voretigene neparvovec [LUXTURNA]
Refer to Section 1.1 of the clinical reviewer memo for information about participation in the clinical trials and any analysis of demographic subgroup outcomes that is notable.
- December 19, 2017 Approval Letter - LUXTURNA
- December 18, 2017 Summary Basis for Regulatory Action - LUXTURNA
- Approval History, Letters, Reviews, and Related Documents - LUXTURNA