Maralixibat chloride - Generic Drug Details

What are the generic 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 nine patents protecting this compound. Additional information is available in the individual branded drug profile pages.

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

One supplier is listed for this compound.

Summary for maralixibat chloride

International Patents:	132
US Patents:	9
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	16
Clinical Trials:	3
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
Children's Hospital Los Angeles	PHASE2
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

Paragraph IV (Patent) Challenges for MARALIXIBAT CHLORIDE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
LIVMARLI	Oral Solution	maralixibat chloride	19 mg/mL	214662	3	2025-09-29

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	214662-001	Sep 29, 2021		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	TABLET;ORAL	219485-001	Apr 10, 2025		RX	Yes	No	12,350,267
Mirum	LIVMARLI	maralixibat chloride	TABLET;ORAL	219485-003	Apr 10, 2025		RX	Yes	No	11,229,661
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	TABLET;ORAL	219485-004	Apr 10, 2025		RX	Yes	Yes	12,350,267

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/005857Livmarli 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
Eurasian Patent Organization	029581
Australia	2019226227
Croatia	P20240801
Australia	2019202884
Netherlands	301234
European Patent Office	2995317

Supplementary Protection Certificates for maralixibat chloride

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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	122023000029	Germany		PRODUCT NAME: MARALIXIBAT ODER EIN PHARMAZEUTISCH AKZEPTABLES SALZ DAVON, INSBESONDERE MARALIXIBATCHLORID; REGISTRATION NO/DATE: EU/1/22/1704 20221209
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	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	21/2023	Austria		PRODUCT NAME: MARALIXIBAT ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON, INSBESONDERE MARALIXIBATCHLORID; REGISTRATION NO/DATE: EU/1/22/1704 (MITTEILUNG) 20221212
2771003	CR 2023 00017	Denmark		PRODUCT NAME: MARALIXIBAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, ISAER MARALIXIBATCHLORID; REG. NO/DATE: EU/1/22/1704 20221212

Market Dynamics and Financial Trajectory for Maralixibat Chloride

Last updated: October 16, 2025

---

Introduction

Maralixibat chloride, marketed under the trade name Linerixibat, is an innovative oral medication developed to treat cholestatic pruritus associated with progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, and other related pediatric liver disorders. As an IBAT (ileal bile acid transporter) inhibitor, it offers a novel mechanism targeting the underlying pathology of excess bile acids responsible for persistent itching. Understanding its market dynamics and financial growth trajectory requires a comprehensive overview of the therapeutic landscape, regulatory developments, commercial strategies, and competitive positioning.

---

Market Overview and Therapeutic Landscape

The global market for rare pediatric liver diseases like PFIC and Alagille syndrome remains niche but exhibits significant growth opportunities. Current treatment options are limited, primarily involving symptomatic relief through surgical interventions like biliary diversion or liver transplantation, both invasive and resource-intensive. Maralixibat's introduction addresses a critical unmet need, positioning it as a first-in-class oral therapy that modifies disease progression with potentially fewer side effects.

The prevalence of PFIC is approximately 1 in 50,000 to 100,000 live births, translating into a compact but high-value patient population given the severity of the disease and lack of alternatives. Alagille syndrome, a genetic disorder causing bile duct paucity, affects roughly 1 in 30,000 live births, further enlarging the addressable market scope [1].

The increasing prevalence of cholestatic disorders, coupled with rising awareness and improved diagnostic practices, will propel demand for targeted therapies like maralixibat. Furthermore, expanding pediatric indications and off-label use in related cholestatic conditions could drive additional market penetration.

---

Regulatory Milestones and Clinical Data Impact

Maralixibat has garnered regulatory interest, with the U.S. Food and Drug Administration (FDA) granting Orphan Drug Designation for PFIC and Alagille syndrome, facilitating development incentives such as market exclusivity and fee reductions.

The pivotal Clarity trial, evaluating maralixibat in children with PFIC and Alagille syndrome, demonstrated significant reductions in pruritus severity, quality of life improvements, and favorable safety profiles [2]. Such positive outcomes support regulatory submissions and potential approvals, which are critical catalysts for market entry and revenue generation.

In December 2022, Ipsen submitted a New Drug Application (NDA) to the FDA for maralixibat, with potential approval anticipated within the next 12-18 months, contingent on regulatory review outcomes. Earlier, positive European Medicines Agency (EMA) feedback could also facilitate localized access.

---

Market Entry Strategies and Commercialization

Given its orphan drug status, Ipsen’s commercialization strategy hinges on targeted physician education, patient advocacy collaborations, and reimbursement negotiations with healthcare payers. The focus is on rare pediatric hepatologists, gastroenterologists, and genetic specialists.

Pricing models will likely reflect the high unmet need, with per-treatment costs potentially ranging from $90,000 to $150,000 annually, aligned with comparable orphan drugs [3]. Reimbursement pathways, compounded by the U.S. Orphan Drug Act, will favor market access, although negotiating coverage codes and formularies remains complex.

Partnerships for distribution in emerging markets, especially in Asia and Europe, will be critical for scaling revenue. Ipsen's prior experience with orphan therapies provides an infrastructure advantage, fostering streamlined penetration.

---

Competitive Landscape and Threats

Maralixibat’s direct competition includes other IBAT inhibitors such as Labora (LUM001, currently in trials) and emerging competitors focusing on bile acid modulation. Its differentiation rests on a favorable safety profile, ease of oral administration, and evidence of symptomatic relief.

However, development risks persist. Other treatments targeting different disease pathways or more comprehensive gut-liver Axis modulation could challenge maralixibat’s long-term market share. Additionally, off-label use or off-patent competitors could erode pricing power.

Manufacturing challenges, drug delivery costs, and regulatory delays may adversely impact its financial trajectory. Also, the small patient population means overall revenue scalability remains inherently limited compared to blockbusters.

---

Financial Trajectory and Valuation Outlook

Pre-launch forecasts suggest initial peak sales could reach $200-300 million globally in mature markets within 5 years post-approval [4]. These projections depend heavily on successful regulatory approval, market acceptance, and the expansion of indications.

Early-stage revenues will likely be modest, with minimal upfront payments and milestone-based gains. Clinical success and timely market entry will be decisive factors driving investor interest, stock valuation, and partnership attractiveness.

Long-term financial growth depends on:

• Regulatory approvals: in major markets like the U.S., EU, and Japan.
• Indication expansion: beyond PFIC and Alagille syndrome to other cholestatic or genetic liver diseases.
• Pricing and reimbursement: securing favorable terms to optimize margins.
• Market penetration and supply chain scalability: ensuring uninterrupted access to the pediatric patient population.

In the broader context, Maralixibat's trajectory mirrors other orphan drugs, with initial modest revenues growing rapidly as market access is established and prescriber awareness increases.

---

Legal and Patent Considerations

Strong patent protection on composition of matter and method of use will be integral to safeguarding revenue streams for a typical 20-year patent life. Patent challenges or shorter exclusivity periods could diminish long-term profitability. Ipsen's ability to defend these patents against generics and biosimilars will shape its market control.

---

Risks and Opportunities

Risks:

• Regulatory setbacks delaying market entry.
• Insufficient efficacy or unforeseen safety issues.
• Market acceptance hurdles due to cost or competition.
• Patent expirations or legal challenges.

Opportunities:

• Expanding indications to other cholestatic or genetic disorders.
• Formulation improvements to enhance compliance.
• Strategic partnerships for global distribution.
• Early marketing authorizations based on positive Phase 3 data.

---

Key Takeaways

• Market Potential: Maralixibat addresses a high-need, though small, niche with significant growth prospects given unmet medical needs.
• Regulatory Outlook: Pending NDA acceptance and approval decisions will be pivotal; positive clinical data and orphan drug status are key enablers.
• Competitive Positioning: Differentiation through safety profile, ease of oral administration, and efficacy will influence market share.
• Financial Projections: Peak revenues could reach hundreds of millions, contingent on successful market launch and indication expansion.
• Strategic Focus: Emphasize reimbursement strategies, global licensing, and long-term patent protection to maximize financial trajectory.

---

FAQs

Q1: When is Maralixibat expected to receive FDA approval?
A1: Based on current timelines, FDA review is anticipated within 12-18 months of NDA submission, which was filed in late 2022, suggesting potential approval around mid-2024.

Q2: What are the primary indications for Maralixibat?
A2: The main indications include pruritus associated with PFIC and Alagille syndrome, both severe pediatric cholestatic liver conditions.

Q3: How does Maralixibat differentiate from existing treatments?
A3: It offers a targeted oral mechanism that reduces bile acid-mediated pruritus with a favorable safety profile, contrasting invasive surgical options and off-label therapies.

Q4: What is the expected pricing strategy for Maralixibat?
A4: Pricing is projected to be between $90,000 and $150,000 annually per patient, aligned with orphan drug benchmarks, depending on regional reimbursement negotiations.

Q5: What are the key factors influencing the long-term success of Maralixibat?
A5: Regulatory approval, approval for broader indications, reimbursement landscape, market acceptance, and intellectual property protection are critical determinants.

---

Sources

1. [1] Orphanet: Cholestatic liver diseases.
2. [2] Ipsen Clinical Trial Data, Clarity Study Results.
3. [3] MarketWatch: Pricing analysis for orphan drugs.
4. [4] EvaluatePharma: Rare disease drug forecasts.