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News
A new treatment may finally bring relief to patients with primary sclerosing cholangitis (PSC). Phase 2b VISTAS trial data showed that volixibat, an investigational bile acid transporter inhibitor, significantly reduced cholestatic pruritus within just two weeks — with improvements in sleep disturbance and fatigue also reported. There are currently no approved pharmacologic therapies for PSC-related symptoms.
For patients with primary sclerosing cholangitis (PSC) suffering from relentless itching, a new therapy may be on the horizon. Data from the 28-week phase 2b VISTAS trial, presented at the ACG Annual Scientific Meeting, showed that volixibat — an investigational ileal bile acid transporter (IBAT) inhibitor from Mirum Pharmaceuticals — significantly reduced cholestatic pruritus compared to placebo, with relief kicking in within just two weeks of starting treatment.
The trial enrolled 111 patients (aged ≥12 years) with moderate to severe pruritus, randomly assigned to volixibat 20 mg twice daily or placebo. Beyond itch relief, volixibat also significantly improved sleep disturbance and reduced serum bile acid levels, while fatigue scores trended in the right direction. The safety profile was consistent with other IBAT inhibitors, with diarrhea and abdominal pain being the most common side effects.
By the Numbers:
Why it matters: PSC currently has no approved pharmacologic therapies targeting its symptom burden, making this the first controlled evidence of a potential treatment for cholestatic symptoms in this population. If these results hold in larger trials, volixibat could fill a critical unmet need for PSC patients.
