Voretigene neparvovec - Spark Therapeutics

Drug Profile

Voretigene neparvovec - Spark Therapeutics

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

Latest Information Update: 29 Jan 2018

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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

  • Registered Leber congenital amaurosis

Most Recent Events

  • 24 Jan 2018 Spark Therapeutics and Novartis enter into a manufacturing and supply agreement for voretigene neparvovec
  • 24 Jan 2018 Voretigene neparvovec licensed to Novartis worldwide except USA
  • 19 Dec 2017 Registered for Leber congenital amaurosis (In children, In adolescents, In adults) in USA (Intraocular) - first global approval
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