Voretigene neparvovec - Spark Therapeutics

Drug Profile

Voretigene neparvovec - Spark Therapeutics

Alternative Names: AAV2 hRPE65v2; AAV2-RPE65v2; Leber congenital amaurosis gene therapy; LTW 888; LUXTURNA; SPK-RPE65; voretigene neparvovec-rzyl

Latest Information Update: 25 Sep 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

  • Marketed Leber congenital amaurosis

Most Recent Events

  • 24 Sep 2018 The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommends approval of Voretigene neparvovec in the treatment of Retinitis pigmentosa in the EU
  • 07 Aug 2018 Launched for Leber congenital amaurosis (In adolescents, In children, In adults) in USA (Intraocular) before August 2018
  • 24 Jan 2018 Spark Therapeutics and Novartis enter into a manufacturing and supply agreement for voretigene neparvovec
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