The Food and Drug Administration (FDA) has granted Fast Track designation to intravenous choline chloride (Protara Therapeutics), an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure-associated liver disease (IFALD).
Intravenous choline chloride is intended for IFALD patients receiving parenteral nutrition (PN). While the exact mechanism of action is not fully understood, it is believed that the use of exogenous choline supplementation may reverse steatosis through its role in very low-density lipoprotein synthesis.
In late 2018, the Company held an end of phase 2 trial meeting with the FDA. Results from the trial showed that treatment with IV choline chloride led to normalization of plasma-free choline concentrations and improvement of hepatic steatosis, as well as statistically significant improvement in cholestasis.
“We look forward to collaborating with FDA and utilizing the benefits associated with Fast Track designation to make this important therapy available for patients with IFALD,” said Jesse Shefferman, CEO of Protara.
Intravenous choline chloride has also been granted Orphan Drug designation for the treatment of IFALD and for the prevention of choline deficiency in PN patients.
Recently, the Company announced its name change from ArTara Therapeutics to Protara Therapeutics.
For more information visit protaratx.com.