• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • JobWatch
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Vivet’ Vtx 801 for Wilson’s receives European/U.S. Orphan designation

Vivet’ Vtx 801 for Wilson’s receives European/U.S. Orphan designation

September 27, 2017
CenterWatch Staff

Vivet Therapeutics, announced that both the FDA and the European Commission (EC) have granted Orphan Drug Designation (ODD) for Vivet’s lead gene therapy product, VTX-801, for the treatment of Wilson’s disease (WD), a chronically debilitating and life-threatening condition if not treated.

“This designation validates Vivet’s efforts to treat the copper metabolism disorder, WD. The FDA and EC have both recognized the unmet need for a safe and effective treatment of Wilson’s Disease by physiological restoration of copper homeostasis and the potential of VTX 801 to address such need" stated Jean-Philippe Combal, CEO of Vivet Therapeutics.

VTX-801 is Vivet’s first gene therapy product to be granted ODD. This is an important value driver for Vivet, in particular through the 10 & seven-years market exclusivity in the EU & U.S. respectively if VTX-801 is approved for the treatment of WD. Initiation of clinical development is planned for Q4 2018 in both the U.S. and the EU.

“Through our strategic collaboration with Fundación para la Investigación Médica Aplicada (FIMA) at the Centro de Investigación Medica Aplicada (University of Navarra, Spain), we are building an unparalleled platform to address key challenges in gene therapy, notably improving sustainability and immune tolerance, targeting Wilson’s disease and other hepatic rare metabolic genetic disorders. A critical asset to this platform is a new liver targeting AAV-Anc80 serotype, licensed from Massachusetts Eye and Ear (MEE), a teaching hospital of Harvard Medical School, Boston" added Jean-Philippe Combal.

Upcoming Events

  • 28Jun

    Effective Root Cause Analysis and CAPA Investigations for the Life Sciences

  • 16Oct

    WCG MAGI's Clinical Research Hybrid Conference - 2022 West

Featured Products

  • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

  • Surviving an FDA GCP Inspection

    Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

Featured Stories

  • Protocol-360x240.png

    Avoid Deviations by Making Protocol Review a Team Effort

  • SelectionProcess-360x240.png

    Give Us a Voice: Sites Clamor for a Say on Vendor Selection

  • Convince-360x240.png

    Use Data and Details to Convince Site Leadership to Add Staff

  • AsktheExpertsBadge-360x240.png

    Ask the Experts: Listing Trial Staff and Others on the Statement of Investigator

Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

The information you need to adapt your monitoring plan to changing times.

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy
  • Do Not Sell My Personal Information

Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2022. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing