A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]

Trial Profile

A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]

Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 12 Oct 2017

At a glance

  • Drugs Voretigene neparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Registrational; Therapeutic Use
  • Sponsors Spark Therapeutics
  • Most Recent Events

    • 12 Oct 2017 According to a Spark Therapeutics media release, the U.S. Food and Drug Administration's (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of LUXTURNA™ (voretigene neparvovec), an investigational, potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).
    • 31 Jul 2017 According to a Spark Therapeutics media release, the company has submitted a Marketing Authorization Application (MAA) to the EMA for LUXTURNA for the treatment of patients with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations.
    • 20 Jul 2017 According to a Spark Therapeutics media release, FDA's Office of Tissues and Advanced Therapies currently is reviewing the BLA for LUXTURNA under a Priority Review designation with a Prescription Drug User Fee Act (PDUFA) action date of Jan. 12, 2018.
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