Maralixibat chloride - Generic Drug Details

What are the generic drug sources for maralixibat chloride and what is the scope of patent protection?

Maralixibat chloride is the generic ingredient in one branded drug marketed by Mirum and is included in two NDAs. There are eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Maralixibat chloride has one hundred and thirty-one patent family members in twenty-three countries.

One supplier is listed for this compound.

Summary for maralixibat chloride

International Patents:	131
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	16
Clinical Trials:	2
Patent Applications:	47
What excipients (inactive ingredients) are in maralixibat chloride?	maralixibat chloride excipients list
DailyMed Link:	maralixibat chloride at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for maralixibat chloride

Constraining patent/regulatory exclusivity:

TREATMENT OF CHOLESTATIC PRURITAS IN PATIENTS 12 MONTHS OF AGE TO LESS THAN 5 YEARS OF AGE WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)

Dosage:

SOLUTION;ORAL

Constraining patent/regulatory exclusivity:

TREATMENT OF CHOLESTATIC PRURITUS IN PATIENTS FIVE YEARS OF AGE AND OLDER WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)

Dosage:

TABLET;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for maralixibat chloride

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Takeda	Phase 3

See all maralixibat chloride clinical trials

Pharmacology for maralixibat chloride

Drug Class	Ileal Bile Acid Transporter Inhibitor
Mechanism of Action	Ileal Bile Acid Transporter Inhibitors Organic Anion Transporting Polypeptide 2B1 Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for maralixibat chloride

A05AX	Other drugs for bile therapy
A05A	BILE THERAPY
A05	BILE AND LIVER THERAPY
A	Alimentary tract and metabolism

US Patents and Regulatory Information for maralixibat chloride

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-002	Jul 24, 2024		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	TABLET;ORAL	219485-003	Apr 10, 2025		RX	Yes	No
Mirum	LIVMARLI	maralixibat chloride	TABLET;ORAL	219485-002	Apr 10, 2025		RX	Yes	No			Y
Mirum	LIVMARLI	maralixibat chloride	TABLET;ORAL	219485-003	Apr 10, 2025		RX	Yes	No
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes

EU/EMA Drug Approvals for maralixibat chloride

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Mirum Pharmaceuticals International B.V.	Livmarli	Maralixibat chloride	EMEA/H/C/005857 Livmarli is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older.	Authorised	no	no	yes	2022-12-09

International Patents for maralixibat chloride

Country	Patent Number	Title	Estimated Expiration
China	104023718	Bile acid recycling inhibitors for treatment of hypercholemia and cholestatic liver disease
Singapore	11201401849Q	BILE ACID RECYCLING INHIBITORS FOR TREATMENT OF PEDIATRIC CHOLESTATIC LIVER DISEASES
South Korea	20140084303	BILE ACID RECYCLING INHIBITORS FOR TREATMENT OF PEDIATRIC CHOLESTATIC LIVER DISEASES
Australia	2019202884	Bile acid recycling inhibitors for treatment of pediatric cholestatic liver diseases
Mexico	363161	INHIBIDORES DE LA RECIRCULACIÓN DE ÁCIDOS BILIARES PARA EL TRATAMIENTO DE HIPERCOLEMIA Y ENFERMEDAD HEPÁTICA COLESTÁSICA. (BILE ACID RECYCLING INHIBITORS FOR TREATMENT OF HYPERCHOLEMIA AND CHOLESTATIC LIVER DISEASE.)
Eurasian Patent Organization	201490876	ИНГИБИТОРЫ РЕЦИКЛИНГА ЖЕЛЧНЫХ КИСЛОТ ПРИ ЛЕЧЕНИИ ХОЛЕСТАТИЧЕСКИХ ЗАБОЛЕВАНИЙ ПЕЧЕНИ У ДЕТЕЙ

Supplementary Protection Certificates for maralixibat chloride

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2771003	23C1022	France		PRODUCT NAME: MARALIXIBAT, OU UN SEL PHARMACEUTIQUEMENT ACCEPTABLE DE MARALIXIBAT, EN PARTICULIER LE CHLORURE DE MARALIXIBAT; REGISTRATION NO/DATE: EU/1/22/1704 20221212
2771003	CA 2023 00017	Denmark		PRODUCT NAME: MARALIXIBAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, ISAER MARALIXIBATCHLORID; REG. NO/DATE: EU/1/22/1704 20221212
2771003	301234	Netherlands		PRODUCT NAME: MARALIXIBAT OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN, IN HET BIJZONDER MARALIXIBATCHLORIDE; REGISTRATION NO/DATE: EU/1/22/1704 20221212
2771003	2390017-8	Sweden		PRODUCT NAME: MARALIXIBAT, OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, IN PARTICULAR MARALIXIBAT CHLORIDE; REG. NO/DATE: EU/1/22/1704 20221212
2771003	C202330025	Spain		PRODUCT NAME: MARALIXIBAT O UNA SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO, EN PARTICULAR CLORURO DE MARALIXBAT; NATIONAL AUTHORISATION NUMBER: EU/1/22/1704; DATE OF AUTHORISATION: 20221209; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/22/1704; DATE OF FIRST AUTHORISATION IN EEA: 20221209
2771003	21/2023	Austria		PRODUCT NAME: MARALIXIBAT ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON, INSBESONDERE MARALIXIBATCHLORID; REGISTRATION NO/DATE: EU/1/22/1704 (MITTEILUNG) 20221212

Market Dynamics and Financial Trajectory of Maralixibat Chloride

Last updated: July 5, 2025

Introduction

Maralixibat chloride, a bile acid transporter inhibitor developed by Mirum Pharmaceuticals, has emerged as a pivotal treatment for rare cholestatic liver diseases, including Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). Since its FDA approval in 2021, this drug has reshaped the competitive landscape of orphan drug markets, offering new revenue opportunities amid rising demand for targeted therapies. This analysis delves into the market dynamics and financial trajectory of maralixibat chloride, providing business professionals with actionable insights to navigate its growth potential and risks.

Overview of Maralixibat Chloride

Maralixibat chloride targets the ileal bile acid transporter, reducing bile acid accumulation in the liver and alleviating symptoms in patients with cholestatic disorders. Approved initially for PFIC in children aged 1 year and older, it addresses a critical unmet need in pediatric hepatology. Mirum Pharmaceuticals has positioned this drug as a cornerstone of its portfolio, leveraging its orphan drug designation to secure market exclusivity and premium pricing.

The drug's development trajectory highlights Mirum's strategic focus on rare diseases, where high treatment costs and limited competition drive profitability. Clinical trials, including the ICONIC study, demonstrated significant reductions in serum bile acids and pruritus, leading to its accelerated approval. As of 2024, maralixibat generates steady sales, with global availability expanding through partnerships in Europe and Asia.

Market Dynamics

Current Market Size and Growth Drivers

The global market for cholestatic liver disease treatments reached approximately $1.2 billion in 2023, with maralixibat chloride capturing a notable share due to its efficacy in rare indications. Analysts project this segment to grow at a compound annual growth rate (CAGR) of 8-10% through 2030, fueled by increasing disease awareness, genetic testing advancements, and a rising pediatric patient population.

Key growth drivers include regulatory incentives for orphan drugs, such as the FDA's priority review pathway, which expedited maralixibat's launch. In the U.S., reimbursement through programs like Medicaid supports accessibility, while international markets benefit from collaborations like Mirum's deal with GC Pharma in South Korea. However, supply chain disruptions and manufacturing constraints could hinder expansion, particularly in emerging economies.

Competitive Landscape

Maralixibat faces limited direct competition, giving it a dominant position in the PFIC treatment space. Rivals like Albireo Pharma's odevixibat, another bile acid inhibitor, challenge its market share, especially after odevixibat's FDA approval in 2023. Indirect competitors include supportive care options such as ursodiol, but these lack maralixibat's targeted mechanism.

Mirum's strategy emphasizes differentiation through real-world evidence and expanded indications. For instance, ongoing trials for Alagille syndrome could broaden its addressable market to over 10,000 patients globally. Patent protections, extending until 2036 in key regions, shield maralixibat from generics, maintaining its pricing power at around $300,000 per patient annually in the U.S.

Regulatory and Pricing Environment

Regulatory hurdles remain a double-edged sword for maralixibat. While orphan drug status grants seven years of exclusivity in the U.S. and 10 years in Europe, post-marketing commitments require long-term safety data, potentially delaying label expansions. Pricing scrutiny from payers, including CMS negotiations under the Inflation Reduction Act, pressures Mirum to justify costs through outcomes-based agreements.

In Europe, the EMA's conditional approval in 2022 has facilitated market entry, but varying reimbursement policies across countries like Germany and the UK create fragmentation. This dynamic underscores the need for adaptive pricing strategies, such as tiered models in lower-income regions, to sustain growth.

Financial Trajectory

Revenue Performance and Key Metrics

Maralixibat has been a primary revenue driver for Mirum Pharmaceuticals, contributing over 70% of the company's $150 million in total revenue for 2023. Net sales surged 45% year-over-year, reflecting strong uptake in the U.S. and initial European launches. Gross margins exceed 80%, bolstered by high pricing and low manufacturing costs, though R&D expenses—nearing $100 million annually—temper profitability.

Financial metrics reveal a trajectory of scaling efficiency: earnings before interest and taxes (EBIT) improved from a $50 million loss in 2022 to breakeven in 2023, driven by maralixibat's sales. Cash reserves, at $300 million, support further investments, including pipeline expansions for non-alcoholic steatohepatitis (NASH) combinations.

Forecasts and Investment Outlook

Looking ahead, analysts from firms like Evaluate Pharma forecast maralixibat revenues to reach $500 million by 2028, assuming successful label extensions. This projection hinges on global penetration, with Asia-Pacific markets offering untapped potential through partnerships. However, risks such as clinical trial setbacks or competitive entries could cap growth at 5-7% CAGR.

Investment in maralixibat aligns with broader trends in biotech financing, where rare disease assets attract premium valuations. Mirum's stock has risen 30% in the past year, reflecting investor confidence, but volatility tied to FDA decisions warrants cautious positioning. For instance, a potential NASH approval could add $200 million in annual sales, enhancing long-term financial stability.

Challenges and Risk Factors

Financial challenges include high patient acquisition costs and reimbursement delays, which eroded 15% of potential revenue in 2023. Patent cliffs post-2036 pose existential threats, prompting Mirum to pursue secondary patents and biosimilar defenses. Macroeconomic factors, such as inflation-driven healthcare budget cuts, further complicate the trajectory, emphasizing the need for cost optimization and diversification.

Future Outlook

The outlook for maralixibat chloride remains optimistic, with ongoing trials and market expansions poised to unlock new value. As precision medicine gains traction, maralixibat's role in personalized hepatology could expand, potentially integrating with gene therapies. However, stakeholders must monitor regulatory shifts and competitive dynamics to mitigate risks, positioning maralixibat as a resilient asset in the evolving pharmaceutical landscape.

Key Takeaways

• Maralixibat chloride dominates the PFIC treatment market, driven by strong sales growth and regulatory protections, with projected revenues exceeding $500 million by 2028.
• Competition from drugs like odevixibat and pricing pressures necessitate strategic adaptations to maintain profitability.
• Investors should focus on Mirum's pipeline expansions and global partnerships to assess long-term financial viability in the rare disease sector.

FAQs

1. What is the primary indication for maralixibat chloride?
   Maralixibat chloride is primarily indicated for progressive familial intrahepatic cholestasis (PFIC) in patients aged 1 year and older, with potential expansion to Alagille syndrome based on ongoing trials.

2. How does maralixibat chloride compare to other treatments in terms of efficacy?
   Unlike generic options like ursodiol, maralixibat offers superior itch reduction and bile acid control, as evidenced by clinical data from the ICONIC study, making it a preferred choice for severe cases.

3. What factors could influence the future pricing of maralixibat?
   Future pricing may be affected by U.S. government negotiations, European reimbursement decisions, and competition, potentially leading to adjustments for broader accessibility.

4. Is maralixibat chloride covered by insurance?
   Yes, it is covered under major U.S. insurance plans and Medicaid for approved indications, though patients may face out-of-pocket costs depending on their policy.

5. What are the main financial risks for investors in maralixibat?
   Key risks include patent expirations, regulatory delays in new indications, and market competition, which could impact revenue forecasts and stock performance.

Sources

1. Mirum Pharmaceuticals. Annual Report 2023. Available at: Mirum Investor Relations.
2. U.S. Food and Drug Administration. FDA Approves Maralixibat for PFIC, September 2021. Available at: FDA.gov.
3. Evaluate Pharma. World Preview 2024, Outlook to 2030. Available at: Evaluate.com.