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.