Mirum Pharmaceuticals Reports Positive Data for Rare Liver Disease Pipeline at EASL 2026
Event summary
- Mirum Pharmaceuticals presented Phase 2b data for volixibat in PSC and brelovitug in HDV at EASL 2026, alongside LIVMARLI data in PFIC.
- Volixibat showed statistically significant reductions in pruritus and bile acid levels in PSC patients.
- Brelovitug achieved 100% virologic response in HDV patients with advanced disease in the 300 mg QW arm.
- LIVMARLI demonstrated improved event-free survival in PFIC patients compared to real-world data.
- Mirum plans NDA submission for volixibat in H2 2026 and BLA submission for brelovitug in 2027.
The big picture
Mirum's data presentations at EASL 2026 underscore its focus on rare liver diseases with significant unmet needs. The positive results for volixibat and brelovitug position the company for potential regulatory milestones in 2026 and 2027, while LIVMARLI's long-term data strengthens its market position in PFIC. The rare disease space continues to attract investment, with Mirum competing alongside other specialized biopharmaceutical companies in developing targeted therapies.
What we're watching
- Regulatory Pathway
- Whether the FDA will accept the volixibat NDA submission based on the Phase 2b data and the planned pre-NDA meeting.
- Clinical Efficacy
- The pace at which brelovitug can demonstrate sustained efficacy in the Phase 3 AZURE program, particularly in advanced HDV patients.
- Market Opportunity
- How Mirum positions LIVMARLI for expanded use in PFIC and other cholestatic diseases based on long-term data.