U.S. FDA grants Versantis Orphan Drug Designation

Inborn errors of metabolism (IEM) causing hyperammonemia (HA) comprise a group of rare genetic metabolic diseases. A defect in an enzyme blocks the metabolic clearance of ammonia from the blood. This leads to a toxic accumulation of ammonia in the body (hyperammonemia), which affects the brain by causing cerebral edema and severe neurologic impairment through various mechanisms. The most relevant group of inherited genetic defects associated with HA are Urea Cycle Disorders (UCDs) and organic acidemia. Currently, most patients are first diagnosed at clinical presentation when HA can rapidly progress to coma and death if not treated immediately.

With a pipeline of drug and diagnostic products covering chronic and acute indications, Zurich based Versantis seeks to revolutionize the current standard of care for patients suffering from acquired and genetic hepatic deficiencies. Versantis’ lead program, VS-01, is in clinical development as first-line therapy for the timely reversal of ACLF. It harnesses Versantis’ proprietary scavenging liposomes to extract toxins from the body. If approved, it will be the first drug to take advantage of the intraperitoneal route to simultaneously support the liver, kidney, and brain, the organs that most often fail in cirrhotic patients.

VS-01 has received the Orphan drug designation from the European Medicines Agency (EMA) in the acute liver failure indication and the U.S. Food and Drug Administration (FDA) for the treatment of IEM, and a Rare Pediatric Diseases Designation from the U.S. FDA. These regulatory incentives will help to streamline the company's clinical development pathway.

“This orphan drug designation from the FDA validates the scientific rationale of VS-01 in a new indication encompassing multiple genetic disorders where there is a vital need to remove the life-threatening build-up of ammonia,” said Meriam Kabbaj, Chief Operations Officer and Co-founder of Versantis. “VS-01 has now received U.S. FDA ODD for two main indications, hyperammonemia in inborn errors of metabolism and Acute-on-chronic Liver Failure (ACLF). Orphan drug incentives considerably strengthen the clinical development plan of our liposomal scavenging platform technology and provide a huge opportunity soon to address the growing burden of metabolic / liver diseases.”

Positive results from Phase 1b clinical trials
“VS-01 is potentially a game-changing medication for the treatment of acute hyperammonemia in an emergency setting. The new designation from the FDA continues to validate our approach and development of VS-01, which was shown to be safe and well tolerated in our Phase 1b clinical study in patients with decompensated liver cirrhosis,” said Vincent Forster, Versantis’ Co-founder and CEO. “Building on this encouraging first experience in patients, we are in the process of raising new funds to continue advancing VS-01 towards its much-needed use in clinical practice.”

Press release/RAN)