Designation qualifies Versantis to receive FDA priority review voucher (PRV) upon marketing approval of VS-01
Zurich, Switzerland, October 19th, 2020 – Versantis AG, a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation (RPDD) to its lead product candidate VS-01, an ammonia clearance enhancer, for the treatment of Urea Cycle Disorders (UCD). UCD is a rare and life-threatening condition caused by an inherited inborn error of metabolism. Current 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 Co-founder of Versantis. “It is valuable recognition that will help Versantis to ramp up its pediatric program by fostering collaborations with key opinion leader and patients’ associations.”
The FDA grants rare pediatric disease designation for serious or life-threatening diseases primarily affecting children from birth to 18 years and affecting fewer than 200,000 people in the USA. Upon approval of a new drug application, the RPDD renders sponsor companies 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. This voucher may be sold or transferred to another sponsor.
Versantis recently completed a $16M Series B financing round and is currently raising new funds to fuel VS-01 clinical development in rare indications, including UCD.
“This recognition acknowledges the unmet medical needs of children with UCD. Importantly, with this designation, we may qualify to receive a priority review voucher from FDA, at the time of marketing approval of VS-01. It provides significant value to our current effort to develop effective treatments for this devastating rare disease,” said Vincent Forster, Versantis’ Co-founder and CEO.
VS-01 is an innovative liposomal-based 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. With its rapid onset, VS-01 could be the first-line medication of choice for acute hyperammonemia in an emergency setting. VS-01 is currently being evaluated in clinical trials in decompensated cirrhotic patients.