Mirum NDA for Maralixibat Accepted for Review

FDA has accepted for priority review a Mirum Pharmaceuticals NDA for maralixibat, an oral apical sodium dependent bile acid transporter inhibitor for treating cholestatic pruritus in patients with Alagille syndrome, a rare liver disease for which there are currently no approved therapies. The company says the drug works by inhibiting the apical sodium dependent bile acid transporter, “resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications.” The agency has set a user fee review action target date of 9/29.

The maralixibat submission is based on data from the Phase 2b ICONIC study. In the trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term, Mirum says.