Mirum Pharmaceuticals granted Breakthrough Therapy Designation for volixibat

Mirum Pharmaceuticals announced that the FDA has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis, or PBC. The regulatory designation is based on the interim analysis of the Phase 2b VANTAGE study which showed statistically significant improvement versus placebo in pruritus for patients treated with volixibat. The confirmatory portion of the study is ongoing with completion of enrollment expected in 2026.