Gene Therapy Gets Orphan Drug Designation in US and Europe

Mathew Shanley

This morning, Paris-based biotech Vivet Therapeutics announced that its lead gene therapy product VTX-801 has been granted Orphan Drug Designation by both the U.S. Food and Drug Administration (FDA) and the European Commission (EC) for the treatment of Wilson’s Disease.

Wilson’s disease is a genetic disorder in which a mutation prohibits the patient from properly eliminating copper from vital organs such as the liver and brain. The gene therapy is intended to restore copper metabolism via liver cell targeting.

In Wilson’s disease patients, copper accumulation begins at birth but indicators often do not start to present until later in life. Signs vary depending on which parts of your body are affected by the condition, and can include hepatic symptoms (e.g., cirrhosis and acute liver failure),  neurological symptoms (e.g., insomnia and seizures),  and psychiatric symptoms ( e.g., depression and psychosis). Other symptoms may include pancreatitis and menstrual irregularities, among others.

“This designation validates Vivet’s efforts to treat the copper metabolism disorder, Wilson’s Disease. 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 in a press release.

Current treatment options include zinc dosages to prevent the absorption of copper and, in more severe cases, liver transplant is an option. Penicillamine (Cuprimine, Depen) and Trientine (Syprine) are both FDA-approved for the removal of excess copper.

“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 Combal.

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