What Mirum Pharmaceuticals does

Mirum Pharmaceuticals is a commercial-stage biopharmaceutical company focused exclusively on rare diseases, primarily in hepatology and adjacent ultra-rare genetic conditions. Founded in 2018 and headquartered in Foster City, California, the company has built a portfolio of three FDA-approved medicines (LIVMARLI/maralixibat, CHOLBAM/cholic acid, and CTEXLI/chenodiol) targeting cholestatic liver diseases such as Alagille syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC), bile acid synthesis disorders, peroxisomal disorders, and cerebrotendinous xanthomatosis. Mirum's underlying technology centers on ileal bile acid transporter (IBAT) inhibition, monoclonal antibodies, and small-molecule inhibitors of ALK2 and PDE4D, applied to conditions with profound disease burden and no approved alternatives.

The company generates revenue primarily through direct sales of approved medicines, distributed through specialty pharmacy channels in the United States and Canada and through wholly-owned subsidiary offices across Germany, France, Italy, Switzerland, Belgium, the Netherlands, and Austria, with Japan commercialization handled by Takeda. LIVMARLI, an oral IBAT inhibitor available in both liquid and tablet formulations, is the flagship product and the primary revenue driver, with sales reaching $359 million in 2025 (+69% YoY) across 30+ countries. Pricing is managed through specialty pharmacy and patient support programs (Mirum Access Plus), with detailed list pricing not publicly disclosed.

Mirum's growth strategy combines organic label expansions with inorganic deal-making. The January 2026 acquisition of Bluejay Therapeutics added brelovitug, a Breakthrough-Therapy-designated monoclonal antibody for chronic hepatitis delta virus, and the April 2026 exclusive license of zilurgisertib from Incyte brought in an oral ALK2 inhibitor for fibrodysplasia ossificans progressiva with a September 26, 2026 PDUFA date. The company supports its portfolio with a deep medical affairs footprint, extensive patient advocacy partnerships, and a proprietary Scout by Mirum diagnostic testing program that helps identify patients with rare genetic conditions. As of mid-2026, Mirum trades on NASDAQ under MIRM, employs 251-500 staff, and has a market capitalization approaching $7.6 billion following 147% trailing stock appreciation.

Name

Mirum Pharmaceuticals

Legal name

Mirum Pharmaceuticals, Inc.

Website

https://mirumpharma.com

Company type

Public

Founded year

2018

Operating status

Operating

Headcount range

251–500 employees

Short description

Mirum Pharmaceuticals is a commercial-stage biopharmaceutical company that develops and commercializes therapies for rare liver and genetic diseases, serving patients with conditions like Alagille syndrome, PFIC, and hepatitis delta across 30+ countries.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Rare Gastroenterology & Hepatology Disorder Therapies (HLAIAIAL)

akta.pro secondary industries

Specialty Care Pharmaceuticals (HLAIAAAB), Gastroenterology & Hepatology Pharmaceuticals (HLAIAAAI), Infectious Disease Specialty Pharmaceuticals (Non-vaccine) (HLAIACAM)

Keywords

• Rare disease therapeutics
• Specialty pharmaceuticals
• Cholestatic liver treatments
• Monoclonal antibody therapies
• Bile acid modulators

Headquarters

HQ city

Foster City

HQ country

United States

HQ region

North America

Offices6 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Marketing or Sales, Personnel, Operations, Others

Revenue model

1. LIVMARLI (maralixibat) sales: LIVMARLI is the flagship product generating $359 million in 2025 sales (up 69% YoY). Sold in both liquid and tablet formulations for Alagille syndrome and PFIC across U.S., EU, Canada and 30+ countries globally. Revenue from direct commercial sales.
2. CHOLBAM/CTEXLI (bile acid medicines): CHOLBAM (cholic acid) and CTEXLI (chenodiol) generated $161 million in 2025 sales (up 31% YoY). CHOLBAM treats bile acid synthesis disorders and peroxisomal disorders; CTEXLI treats cerebrotendinous xanthomatosis (CTX).
3. Pipeline development and potential future product launches: Mirum expects multiple product launches 2027-2028 including volixibat for PSC/PBC, brelovitug for HDV, and zilurgisertib for FOP, representing future revenue streams from current late-stage pipeline assets.

Go-to-market motion3 records

Distribution channels3 records

Marketing channels5 records

Core offering

Mirum Pharmaceuticals is a commercial-stage biopharmaceutical company that develops and commercializes therapies for rare diseases, primarily cholestatic liver diseases and other ultra-rare conditions. The company markets three FDA-approved medicines — LIVMARLI (maralixibat), CHOLBAM (cholic acid), and CTEXLI (chenodiol) — and has a pipeline of investigational therapies including volixibat, brelovitug, zilurgisertib, and MRM-3379 targeting high-unmet-need patient populations.

Product overview

Mirum Pharmaceuticals is a biopharmaceutical company focused on rare diseases, with three FDA-approved medicines and multiple investigational therapies in its pipeline. The approved portfolio includes LIVMARLI (maralixibat) for Alagille syndrome and PFIC, CHOLBAM (cholic acid) for bile acid synthesis disorders, and CTEXLI (chenodiol) for Cerebrotendinous Xanthomatosis. The pipeline includes volixibat for PSC and PBC, brelovitug for hepatitis delta virus, zilurgisertib for fibrodysplasia ossificans progressiva, and MRM-3379 for Fragile X Syndrome. The company operates as a biopharmaceutical commercialization and development organization rather than a technology platform.

Differentiator

Problem solved

Functional benefit

Brands

• SCOUT by Mirum: Diagnostic testing program partnering with laboratories to offer free testing kits for rare disease diagnosis
• Mirum Access Plus (MAP)
• Embrace Patient Support Program

Products and services

• LIVMARLI (maralixibat) An orally administered ileal bile acid transporter (IBAT) inhibitor approved for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older, and in patients with Progressive Familial Intrahepatic Cholestasis (PFIC) 12 months of age and older. Available in both liquid and tablet formulations.
• CHOLBAM (cholic acid) A bile acid replacement therapy approved for treatment of bile acid synthesis disorders due to single enzyme deficiencies, and for treatment of peroxisomal disorders including Zellweger spectrum disorders in patients showing signs of liver disease.
• CTEXLI (chenodiol) A bile acid therapy approved by the FDA for treatment of adults with Cerebrotendinous Xanthomatosis (CTX). It is the first and only treatment designed to reduce overproduction of toxic bile acid precursors.
• Volixibat A minimally absorbed, orally administered investigational therapy designed to selectively inhibit IBAT for treatment of cholestatic pruritus associated with primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). Has received FDA Breakthrough Therapy designation for PBC.
• Brelovitug An investigational fully human monoclonal antibody being studied for treatment of chronic hepatitis delta virus (HDV), the most severe form of viral hepatitis. Has received FDA Breakthrough Therapy designation and EMA PRIME designation for HDV.
• Zilurgisertib An investigational oral small-molecule therapy designed to selectively inhibit ALK2 (ACVR1) for treatment of fibrodysplasia ossificans progressiva (FOP). Licensed from Incyte for worldwide development and commercialization.
• MRM-3379 An allosteric inhibitor of phosphodiesterase-4D (PDE4D) being studied for treatment of Fragile X Syndrome (FXS). Licensed from Enthorin Therapeutics.

Quantifiable outcome

• LIVMARLI achieved $359 million in 2025 sales, up 69% year-over-year
• +3 more outcomes

Named customers5 records

Segments5 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Nine partnerships are on record, tiered core and minor.

• IncytecoreStrategic or Co-development Partner · 1 April 2026In April 2026, Mirum entered into an exclusive license agreement with Incyte for worldwide rights to zilurgisertib, an oral ALK2 inhibitor for fibrodysplasia ossificans progressiva. Mirum acquired the asset for $16 million upfront plus up to $63 million in milestones. Companies jointly announced positive Phase 2 PROGRESS study data in June 2026.
• Bluejay TherapeuticscoreStrategic or Co-development Partner · 26 January 2026Mirum completed acquisition of Bluejay Therapeutics in January 2026 for $620 million in cash and stock plus up to $200 million in potential milestone payments. Acquired worldwide rights to brelovitug, a late-stage monoclonal antibody for chronic hepatitis delta virus with FDA Breakthrough Therapy designation.
• Davis PolkminorOthers · 1 December 2025Davis Polk advised Mirum on $200 million private placement of common stock and pre-funded warrants to fund clinical development and pipeline expansion.
• TakedacoreChannel Partner/ Reseller/ Distributor · 1 January 2024Takeda serves as commercial partner for LIVMARLI in Japan, handling regulatory approvals and commercialization. LIVMARLI was approved in Japan through partner Takeda for PFIC and Alagille syndrome.
• Enthorin TherapeuticscoreOEM/ Whitelabel/ Licensing Partner · 1 January 2024Mirum licensed MRM-3379 (formerly ENT-3379), an orally available selective PDE4D inhibitor for Fragile X syndrome, from Enthorin Therapeutics. Mirum initiated BLOOM Phase 2 clinical study in December 2025.
• PreventionGeneticsminorStrategic or Co-development Partner · 1 January 2024Partnership through Scout by Mirum program providing free genetic testing kits including 143-gene cholestasis panel, 66-gene early-onset bilateral cataracts panel, and PBD-ZSD testing.
• Cincinnati Children's Hospital Medical CenterminorStrategic or Co-development Partner · 1 January 2024Partnership through Scout by Mirum program providing mass spectrometry testing for bile acid synthesis disorders and peroxisomal disorders at no cost to patients.
• Oregon Health & Science UniversityminorStrategic or Co-development Partner · 1 January 2024Partnership through Scout by Mirum program for cerebrotendinous xanthomatosis (CTX) testing through OHSU Sterol Analysis Clinical Laboratory.
• Kennedy Krieger InstituteminorStrategic or Co-development Partner · 1 January 2024Partnership through Scout by Mirum program for Smith-Lemli-Opitz syndrome (SLOS) testing providing biochemical test for cholesterol and 7/8-DHC intermediates.

Scale indicators10 records

Recent moves6 records

Expansion highlights3 records

Broad incumbents

• Alexion (AstraZeneca Rare Disease): Established rare-disease unit of AstraZeneca with multiple approved rare-disease therapies and global commercial reach. Comparable in commercial rare-disease infrastructure and orphan-drug pricing model.
• Takeda Pharmaceutical: Global biopharma with a large rare disease franchise (post-Shire acquisition) and existing commercial partnership with Mirum in Japan. Operates broadly in rare GI/liver diseases; comparable in therapeutic area but at vastly greater scale.
• Ipsen: Global specialty pharma with strong rare disease and hepatic portfolios. Overlaps with Mirum in liver and rare disease therapy categories but operates at meaningfully greater scale and portfolio breadth.

Direct peers

• Alnylam Pharmaceuticals: Commercial-stage biopharmaceutical company focused on rare genetic diseases with multiple approved RNAi therapeutics. Comparable in scale, rare-disease focus, and reliance on specialty distribution and orphan-drug pricing economics.
• BioMarin Pharmaceutical: Established rare-disease biopharma with a multi-product commercial portfolio of orphan drugs across multiple therapeutic areas. Most directly comparable to Mirum in business model: a focused rare-disease commercial engine with ongoing pipeline replenishment.
• Recordati Rare Diseases: Specialty pharma division focused on rare diseases with global commercialization infrastructure. Comparable in rare-disease commercial model and emphasis on smaller patient populations with high unmet need.
• Ultragenyx Pharmaceutical: Rare and ultra-rare disease biotech with multiple approved products and a similar specialty distribution model. Overlapping customer base (specialty prescribers, rare disease advocacy) and similar pipeline-driven growth narrative.

Emerging players

• Intercept Pharmaceuticals: Liver-focused biopharma commercializing obeticholic acid for PBC. Comparable therapeutic focus on rare cholestatic liver disease and similar commercial model targeting hepatologists, though now under Alfasigma ownership.
• SpringWorks Therapeutics: Commercial-stage rare disease biotech with focused launches in ultra-rare oncology and rare disorders. Comparable in company stage, rare-disease commercial motion, and reliance on single-asset growth narratives.
• Genfit: Late-stage biotech focused on cholestatic and metabolic liver diseases, including PBC. Direct overlap with Mirum's volixibat program in primary biliary cholangitis — a head-to-head competitive threat in the IBAT/PBC space.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/mirum-pharmaceuticals
• Xx.com/mirumpharma
• Facebookfacebook.com/mirumpharma

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Subsidiaries1 record

Funding overview

Funding rounds7 records

Investors11 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Mirum Pharmaceuticals