Clinical stage biotech company Vivet Therapeutics announced on Thursday that it has received Orphan Drug Designation (ODD) from the European Commission for the company's VTX-806, a gene therapy product intended for the treatment of Cerebrotendinous Xanthomatosis (CTX), a rare neurodegenerative disease.

Pre-clinical in vivo studies of the product, an Adeno-associated viral (AAV) vector encoding human CYP27A1, showcased a decrease of hepatomegaly in mice, normalisation of upregulated compensatory enzymes and of primary and secondary bile acids when compared to present standard of care (SOC) treatments. The submitted data, along with the chronically debilitating impacts of CTX associated leukodystrophies, supported the decision by the EU Commission to offer ODD to the product as a potential treatment option.

VTX-806's ODD adoption is subsequent to the EUR4.9m funding that the company received from the French government to progress with the development of the gene therapy for treatment of CTX. The funding is for preclinical research and development activities of the product, including manufacturing process development and a clinical study intended at identifying neurological biomarkers for the effectiveness of treatment in subjects under SOC.

Dr Jean-Philippe Combal, co-founder & chief executive officer at Vivet Therapeutics, said, 'Early diagnosis and treatment of CTX is crucial to halting disease progression. At present, existing standard of care treatments can only slow or stabilize leukodystrophy, with no cure available. Preclinical data supports VTX-806's potential as an alternative treatment option to stop, or reverse, disease progression over the long-term, or possibly even cure CTX patients. Receiving ODD from the EU Commission provides Vivet with certain benefits during development and commercialization activities as we press forward with the anticipation of submitting ODD to the US FDA in 2024 and plan to begin preparations to enter clinical development in late 2025.'