SEFA-6179

SEFA-6179 is a novel, oral, fully synthetic medium chain fatty acid (MCFA) analog developed for the treatment of intestinal failure-associated liver disease (IFALD), also known as parenteral nutrition-associated liver disease (PNALD). It is designed to directly target the liver and has shown beneficial effects in preclinical models by preventing cholestasis and liver injury associated with IFALD. The drug acts as a fatty acid analogue and is intended to preserve the transport characteristics of MCFAs while providing improved pharmacological impact by avoiding rapid metabolism. In Phase 1 clinical trials in healthy volunteers, SEFA-6179 was well tolerated at doses up to 1,000 mg once daily for 14 days; most adverse events were mild or moderate headaches that resolved during treatment. The drug has received FDA Rare Pediatric Disease Designation for IFALD and is currently being evaluated in Phase 2a clinical trials for this indication[1][2][3][4][5][6][8].

Brand names

Orziloben

Other names

OrzilobenNST-6179NST6179NST 6179