Mirum Pharmaceuticals

FDA awards Breakthrough Therapy Designation to Mirum Pharmaceuticals' volixibat for primary biliary cholangitis (PBC), based on Phase 2b VANTAGE study interim results showing significant improvement in cholestatic pruritus. Volixibat, an oral medication inhibiting ileal bile acid transporter (IBAT), aims to reduce systemic bile acids, potentially offering a novel treatment for adult cholestatic diseases. Full VANTAGE study results expected by 2026.

FDA grants Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients, based on positive Phase 2b VANTAGE study results showing significant improvement in pruritus vs. placebo. Enrollment for the confirmatory portion of the study is expected to complete in 2026.

Mirum Pharmaceuticals' volixibat received Breakthrough Therapy Designation for treating cholestatic pruritus in primary biliary cholangitis (PBC) based on VANTAGE study results showing significant pruritus improvement. Volixibat, an oral IBAT inhibitor, aims to reduce bile acids and is being evaluated in phase 2b studies for PBC and primary sclerosing cholangitis.

The European Commission approves Ipsen's IQIRVO (elafibranor) for primary biliary cholangitis, the first new therapy in the EU in a decade. Other companies, including CymaBay, Zydus, and GlaxoSmithKline, are preparing to enter the market. The approval is based on the Phase III ELATIVE trial, showing a 47% clinical benefit of IQIRVO combined with UDCA. Additionally, Gilead Sciences' LIVDELZI (seladelpar) received FDA accelerated approval for PBC.

Mirum's Livmarli approved for PFIC in patients 12 months and older, addressing cholestatic pruritus in rare liver disease.