molecules of the month

The Mirum Pharmaceuticals IBAT bile acid transporter inhibitor, maralixibat, has a long history, having been first patented by scientists at Searle in 1994 and published by the time Searle became part of Pharmacia in 2005. It was finally approved in Sep. 2021 for treatment of cholestatic pruritis in patients with Alagille Syndrome, and is estimated to reach annual sales of $536M by 2026, a nice success story for repurposing of old mechanisms by a biotech. The drug is a bile acid transport inhibitor, preventing enterohepatic bile acid recirculation, and was originally designed to have minimal systemic exposure after oral administration.