Companies To Watch: Mirum Pharmaceuticals

By Wayne Koberstein, Executive Editor, Life Science Leader
Follow Me On Twitter @WayneKoberstein

Two drugs, one FDA approved, together taking on a half-dozen rare liver diseases

SNAPSHOT

Mirum Pharmaceuticals focuses on developing two drugs with multiple indications for rare liver diseases. One of the products, Livmarli (maralixibat), entered the market in 2022 with FDA approval for treating cholestatic pruritus in Alagille syndrome (ALGS). Mirum obtained maralixibat and the other lead drug, volixibat, from Shire to spark its founding in 2018. The company has submitted maralixibat for EU approval of cholestatic liver disease in patients with ALGS as it completes the drug’s Phase 2/3 trials for progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA). It is also in late-stage development of volixibat for primary sclerosing cholangitis (PSC), intrahepatic cholestasis of pregnancy (ICP), and primary biliary cholangitis (PBC).