FDA 授予 Mirum Pharmaceuticals 的 volixibat 突破性疗法认定，用于治疗 PBC 患者的胆汁淤积性瘙痒症。 FDA grants Breakthrough Therapy Designation to Mirum Pharmaceuticals' volixibat for treating cholestatic pruritus in PBC patients.
Mirum Pharmaceuticals 的 volixibat 已被 FDA 授予突破性疗法认定，用于治疗原发性胆汁性胆汁炎 （PBC） 患者的胆汁淤积性瘙痒症。 Mirum Pharmaceuticals' volixibat has been granted Breakthrough Therapy Designation by the FDA for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients. 这项决定的依据是第二阶段b VANTAGE研究的中期积极结果,表明普里图斯和血清丁酸有显著改善。 This decision is based on positive interim results from the Phase 2b VANTAGE study, indicating significant improvements in pruritus and serum bile acids. 正在进行的确认性研究预计到2026年完成入学,促进病人的快速发展和就诊。 The ongoing confirmatory study is expected to complete enrollment by 2026, facilitating expedited development and access for patients.