Voretigene neparvovec - Spark Therapeutics
Alternative Names: AAV2 hRPE65v2; AAV2-RPE65v2; Leber congenital amaurosis gene therapy; LTW 888; LUXTURNA; Luxturna; SPK-RPE65; voretigene neparvovec-rzylLatest Information Update: 05 Nov 2023
At a glance
- Originator The Childrens Hospital of Philadelphia
- Developer Novartis; Spark Therapeutics
- Class Eye disorder therapies; Gene therapies
- Mechanism of Action Gene transference
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Marketed Leber congenital amaurosis; Retinal dystrophies
Most Recent Events
- 30 Aug 2023 Launched for Retinal dystrophies (In adolescents, In children, In adults) in Japan (Intraocular, Injection)
- 26 Jun 2023 Preregistration for Retinal dystrophies (In adolescents, In children, In adults) in Japan, prior to June 2023(Intraocular, Injection)
- 26 Jun 2023 Registered for Retinal dystrophies (In adolescents, In children, In adults) in Japan (Intraocular, Injection)