Voretigene neparvovec

Drug Profile

Voretigene neparvovec

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

Latest Information Update: 21 Jul 2017

Price : $50

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
  • New Molecular Entity No

Highest Development Phases

  • Preregistration Leber congenital amaurosis

Most Recent Events

  • 21 Jul 2017 Planned Prescription Drug User Fee Act (PDUFA) date for Leber congenital amaurosis in USA (Intraocular) is 2018-01-21
  • 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 (12/01/2017) for voretigene neparvovec for Leber congenital amaurosis
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