Voretigene neparvovec

Drug Profile

Voretigene neparvovec

Alternative Names: AAV2 hRPE65v2; Leber congenital amaurosis gene therapy; LUXTURNA; SPK-RPE65

Latest Information Update: 16 Aug 2017

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At a glance

  • Originator The Childrens Hospital of Philadelphia
  • Developer 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.

    Yes - Leber congenital amaurosis; Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Preregistration Leber congenital amaurosis

Most Recent Events

  • 29 Jul 2017 Preregistration for Leber congenital amaurosis in European Union (Intraocular)
  • 20 Jul 2017 Voretigene neparvovec granted Rare paediatric disease designation for Leber congenital amaurosis from the US FDA
  • 17 Jul 2017 FDA assigns PDUFA action date of 12/01/2018 for voretigene neparvovec for Leber congenital amaurosis
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