Voretigene neparvovec

Drug Profile

Voretigene neparvovec

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

Latest Information Update: 24 May 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

  • 18 May 2017 Spark Therapeutics completes rolling BLA submission for Leber congenital amaurosis in USA
  • 09 Jan 2017 Updated efficacy and adverse event data from a phase I trial in Leber congenital amaurosis released by Spark Therapeutics
  • 10 Aug 2016 Updated efficacy and safety data from phase III trial in Leber congenital amaurosis released by Spark Therapeutics
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