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Orphan Drug Designation (ODD) granted to Vonafexor for Alport syndrome by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA)
ENYO Pharma announces that both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted Orphan Drug Designation (ODD) to Vonafexor, our fibrolytic & anti-inflammatory clinical compound, for the treatment of Alport syndrome.
Alport syndrome is a rare kidney disease, a genetic disorder affecting 1 in 5,000-10,000 live births. It is characterized by a progressive loss of kidney function and may result in hearing loss and eye abnormalities.
Other programs
Vonafexor in HBV and HDV
In addition to its strong fibrolytic and anti-inflammatory effect, Vonafexor displays anti-viral properties against Hepatitis B Virus (HBV) and Hepatitis D Virus (HDV).
ENYO Pharma has already conducted 5 clinical studies (3 phases I and 2 phases II) with Vonafexor for HBV indication. Click here for more information
- Radreau et al. (2016): Reciprocal regulation of Farnesoid X Receptor a activity and hepatitis B virus replication in differentiated HepaRG cells and primary human hepatocytes.
Publications
Ratziu et al. (2022)
Hepatic and renal improvements with FXR agonist Vonafexor in individuals with suspected fibrotic NASH. Journal of Hepatology. 78 (3), 479-492.
Non-alcoholic steatohepatitis (NASH) has become a leading cause of chronic liver disease worldwide. Affected patients are also at higher risk of developing chronic kidney disease. There are no approved therapies and only few options to treat this population. The phase IIa LIVIFY trial results show that single daily administration of oral vonafexor,
Clinical studies
Vonafexor is already supported by 9 completed clinical studies: 6 Phases 1 and 3 Phases 2a.
- Phase 2a LIVIFY study: Safety, Tolerability, Pharmacokinetics and Efficacy of EYP001a in Patients With Nonalcoholic Steatohepatitis (NASH).
Results: The phase IIa LIVIFY trial results show that single daily administration of oral vonafexor, an FXR agonist, leads in the short term to a reduction in liver fat, liver enzymes, fibrosis biomarkers, body weight and abdominal circumference,
Pipeline
ENYO Pharma is advancing its pipeline with Vonafexor series for diseases with impaired kidney function as CKD, Alport syndrome, NASH and DKD.
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