Vivet Therapeutics’ VTX-806 Receives the EC’s Orphan Drug Designation for Cerebrotendinous Xanthomatosis (CTX)
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- The EC has granted orphan drug designation to VTX-806, an adeno-associated viral (AAV) vector encoding human CYP27A1, for treating cerebrotendinous xanthomatosis (CTX). ODD application to the US FDA is planned in H2’24, with its clinical trials in 2025
- VTX-806 showed reduced hepatomegaly & normalization of upregulated compensatory enzymes & bile acids in mice compared to SoC. This data formed the basis of designation across the EU
- In addition, Vivet obtained $5.32M (EUR 4.9M) funding from the French government to support VTX-806’s preclinical research, manufacturing & clinical development to identify neurological biomarkers for effective CTX treatment
Ref: Vivet Therapeutics | Image: Vivet Therapeutics
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
