Protara Therapeutics’ (NASDAQ:TARA) IV choline chloride received FDA fast track designation for the treatment of intestinal failure-associated liver disease (IFALD).
IFALD occurs in patients who have been receiving parenteral nutrition for more than six months and is characterized by toxic fat accumulation in the liver cells and damage to the biliary system.
IV choline chloride is a Phase 3-ready investigational phospholipid substrate replacement therapy. The drug candidate has been also granted FDA orphan drug designation for the treatment of IFALD and prevention of choline deficiency in patients receiving parenteral nutrition.
“Receiving fast track designation from the FDA further supports the potential for IV choline chloride to serve as the much-needed first approved therapy for IFALD patients,” Jesse Shefferman, Protara’s CEO, said in a statement.
A Phase 2 trial in IFALD patients dependent on parenteral nutrition demonstrated that treatment with IV choline chloride normalized plasma free choline concentrations, reversed hepatic steatosis, and significantly improved cholestasis.