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A key milestone for Versantis: FDA grants the Rare Pediatric Disease Designation to its lead program


A woman and a man in lab coats in a laboratory
Versantis co-founders Meriam Kabbaj and Vincent Forster


Our portfolio company, Versantis AG (Redalpine Capital III), announced that the U.S. Food and Drug Administration (FDA) has granted its lead candidate VS-01 the designation as rare pediatric disease (RPDD) for the treatment of Urea Cycle Disorders (UCD). 


The rare pediatric disease designation is granted by the FDA for serious or life-threatening diseases primarily affecting children from birth to 18 years and affecting fewer than 200,000 people in the USA. The RPDD renders Versantis eligible for a priority review voucher, which can be redeemed to obtain accelerated FDA review of a drug candidate, in any indication, potentially gaining early market access. Versantis’ lead asset VS-01 is an innovative detoxification therapy that acts as a clearance enhancer for a large spectrum of toxic metabolites accumulated during liver and kidney failures. More specifically, VS-01 clears ammonia from the body, which is the main neurotoxic metabolite and can lead to brain edema. VS-01 is currently being evaluated in clinical trials in decompensated cirrhotic patients.


UCD is a rare and life-threatening condition caused by an inherited inborn error of metabolism and currently available treatment options are associated with poor outcomes. 

“The rare pediatric disease designation highlights the potential of using VS-01 to treat the serious and life-threatening manifestations of UCD,” said Meriam Kabbaj, Chief Operations Officer and Cofounder of Versantis. “It is valuable recognition that will help Versantis to ramp up its pediatric program by fostering collaborations with key opinion leaders and patients’ associations.” 


We congratulate the entire Versantis-Team to this major milestone and look forward to the next steps together.


For additional information, visit Versantis Homepage

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