Mirum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to volixibat. This designation is for volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC).
The regulatory designation is based on a positive interim analysis of the Phase 2b VANTAGE study, which showed statistically significant improvement versus placebo in pruritus for patients treated with volixibat. The Breakthrough Therapy Designation aims to expedite the development and review of drugs intended to treat serious or life-threatening diseases.
This milestone indicates that preliminary clinical evidence suggests volixibat may demonstrate substantial improvement over existing therapies for cholestatic pruritus in PBC. The confirmatory portion of the VANTAGE study is ongoing, with enrollment completion expected in 2026.
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