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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 Dec 2021

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At a glance

  • Originator The Childrens Hospital of Philadelphia
  • Developer Novartis; 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
  • Phase III Retinal dystrophies

Most Recent Events

  • 24 Nov 2020 Phase-III clinical trials in Retinal dystrophies (In adults, In adolescents, In children) in Japan (Intraocular) (NCT04516369)
  • 20 Aug 2020 Novartis plans a phase III trial for Retinal dystrophies (In children, In adolescents, In adults, In the elderly) in September 2020 (NCT04516369)
  • 12 Feb 2020 Voretigene neparvovec will be assessed within the ultra-orphan pathway for Leber congenital amaurosis in Scotland
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