Mirum Pharmaceuticals Charts 2026 Expansion With Alagille Syndrome Treatment Advancement

Mirum Pharmaceuticals has reached a critical juncture heading into 2026, marked by strong financial performance and promising clinical developments. The company’s trajectory suggests meaningful growth opportunities ahead, particularly with its expanding treatment portfolio that includes therapies for Alagille syndrome—a rare genetic liver disease affecting a severely underserved patient population.

Financial Strength and Clinical Momentum

The pharmaceutical company has achieved meaningful financial milestones that underscore its transition from development-stage venture to revenue-generating enterprise. Most significantly, Mirum generated positive cash flow from operations in 2025, a watershed moment for any small-cap biotech company. This financial stability provides crucial runway for the company’s ambitious clinical pipeline while reducing dependence on external capital.

Revenue generation has accelerated notably. Livmarli, Mirum’s flagship product, delivered particularly strong results with net product sales jumping 69% year-over-year to reach $359 million in 2025. The company’s established bile acid replacement therapies, including Cholbam and Ctexli, contributed an additional $161 million, representing 31% year-over-year growth. Management has guided for overall revenue between $630 million and $650 million in 2026, signaling confidence in sustained commercial momentum.

Regulatory Approvals Driving Clinical Credibility

Mirum secured two pivotal FDA approvals in 2025, each expanding the company’s addressable market. In February, the FDA granted clearance for Ctexli to treat cerebrotendinous xanthomatosis (CTX), a rare genetic disorder affecting bile acid metabolism. More significantly for rare disease patients, the agency approved a tablet formulation of Livmarli in April specifically for cholestatic pruritus associated with two serious liver conditions: Alagille syndrome and Progressive Familial Intrahepatic Cholestasis (PFIC).

The Alagille syndrome approval represents particularly meaningful progress, as this genetic condition causes progressive liver disease and significantly impacts quality of life. The availability of an oral tablet formulation offers Alagille syndrome patients a more convenient treatment option compared to earlier formulations, potentially improving patient compliance and outcomes.

Strategic Acquisition Expands Rare Disease Reach

In late January 2026, Mirum completed its acquisition of Bluejay Therapeutics, adding brelovitug to its portfolio. Brelovitug targets chronic hepatitis delta virus (HDV), a severe viral infection for which no approved treatment exists in the United States. This acquisition strengthens Mirum’s focus on underserved rare liver diseases and diversifies its clinical pipeline beyond bile acid disorders.

2026 Catalyst Calendar: What to Watch

The year ahead presents multiple inflection points that could substantially influence the company’s trajectory. The most significant involves volixibat, a candidate drug targeting primary sclerosing cholangitis (PSC)—a progressive liver disease with no approved therapies currently available in the U.S. market. Mirum anticipates announcing Phase 2B clinical trial results in the second quarter of 2026, with potential for accelerated approval filing in the second half of the year. Volixibat holds blockbuster potential if regulatory approval materializes, given the substantial unmet need in the PSC patient population.

Concurrently, Mirum is evaluating volixibat in another indication: primary biliary cholangitis (PBC), the most prevalent cholestatic liver disease globally. Enrollment completion in this study is expected by the second half of 2026.

The Bluejay acquisition also introduces near-term catalysts. Phase 3 clinical results for brelovitug in HDV treatment are anticipated in the second half of 2026. Successful data could support an approval filing in early 2027, potentially creating a new revenue stream for the company.

Additionally, Mirum has accelerated the timeline for a Phase 3 study evaluating Livmarli in pruritus associated with rare cholestatic conditions. The company now expects enrollment completion in the first half of 2026 and plans to report results before year-end, potentially establishing Livmarli in additional patient populations.

The Path Forward

Mirum Pharmaceuticals enters 2026 positioned at a meaningful inflection point, combining established commercial success with a robust clinical pipeline. The company’s demonstrated ability to generate positive cash flow, achieve regulatory approvals—including the meaningful Alagille syndrome indication—and strategically acquire complementary assets suggests a company executing on multiple fronts. The catalyst-rich environment throughout 2026 could validate management’s strategic direction and support continued stock performance.