FDA grants Breakthrough Therapy Designation to Mirum Pharmaceuticals' volixibat for treating cholestatic pruritus in PBC patients.
Mirum Pharmaceuticals' volixibat has been granted Breakthrough Therapy Designation by the FDA for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients. This decision is based on positive interim results from the Phase 2b VANTAGE study, indicating significant improvements in pruritus and serum bile acids. The ongoing confirmatory study is expected to complete enrollment by 2026, facilitating expedited development and access for patients.
October 10, 2024
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