Clinical-stage company Versantis AG reported on Monday the receipt of US Food and Drug Administration (FDA) rare paediatric disease designation (RPDD) for its lead product candidate VS-01 for the treatment of Urea Cycle Disorders (UCD), a rare and life-threatening condition caused by an inherited inborn error of metabolism.

The company said 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. VS-01 is currently being evaluated in clinical trials in decompensated cirrhotic patients.

Recently, the company has completed a USD16m Series B financing round and is currently raising new funds to fuel VS-01 clinical development in rare indications, including UCD.

Upon approval of a new drug application, the FDA's RPDD renders sponsor companies eligible for a priority review voucher, which may be sold, transferred or redeemed to obtain accelerated review of a drug candidate, in any indication, potentially gaining early market access.