FDA accepts New Drug Application for maralixibat for the treatment of cholestatic pruritus in ALGS

The U.S. Food and Drug Administration has accepted a new drug application for maralixibat (Mirum Pharmaceuticals), an oral apical sodium dependent bile acid transporter (ASBT) inhibitor for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older, according to a press release.

“We are extremely pleased that our NDA will move forward in the regulatory review process, bringing maralixibat one step closer to being available for patients with Alagille syndrome,” said Chris Peetz, president and chief executive officer at Mirum in a press release. “With more than 6 years of follow-up data showing durability of response as well as safety, we believe that maralixibat, if approved, would provide a meaningful treatment option that will ultimately reduce the need for liver transplantation.”

Read the full press release here.